Pub Date : 2024-08-24DOI: 10.1186/s13063-024-08384-3
Dimitri Daldegan-Bueno, Carina Joy Donegan, Anna Forsyth, Rachael Louise Sumner, Robin J Murphy, David B Menkes, William Evans, Nicholas Hoeh, Frederick Sundram, Lisa M Reynolds, Rhys Ponton, Alana Cavadino, Todd Smith, Partha Roop, Nathan Allen, Binu Abeysinghe, Darren Svirskis, Mahima Bansal, Suresh Muthukumaraswamy
Background: Major depressive disorder (MDD) poses a significant global health burden with available treatments limited by inconsistent efficacy and notable side effects. Classic psychedelics, including lysergic acid diethylamide (LSD), have garnered attention for their potential in treating psychiatric disorders. Microdosing, the repeated consumption of sub-hallucinogenic doses of psychedelics, has emerged as a self-treatment approach for depression within lay communities. Building upon preliminary evidence and the successful completion of an open-label pilot trial of microdosing LSD for depression (LSDDEP1), this protocol outlines a phase 2b randomised controlled trial (LSDDEP2). The main objective of LSDDEP2 is to assess the modification of depressive symptoms, measured by the Montgomery-Åsberg Depression Rating Scale (MADRS), following a regimen of LSD microdoses versus placebo.
Methods: This is a randomised, double-dummy, triple-blind, active placebo-controlled, parallel groups trial of LSD microdosing in patients meeting DSM-5 criteria for major depressive disorder. Participants will undergo an 8-week LSD microdosing regimen using the titratable MB-22001 formulation taking two doses a week. All doses will be self-administered at home and will be titratable from 4 to 20 μg based on subjective perception and tolerability. In addition to depression symptoms, outcome will include psychiatric and personality inventories, sleep and activity tracking, electroencephalography (EEG), blood biomarkers, semi-structured interviews, and safety (e.g. adverse event, laboratory exam) measures.
Discussion: This study will be the first randomised controlled trial to administer controlled microdoses of LSD for treatment of MDD in participants' naturalistic environment. The measures included are designed to assess the drug's safety, mechanism, and treatment efficacy over placebo in this population. The results of this study will be important for assessing the viability of psychedelic microdosing as an additional treatment option and for informing the direction of future clinical trials.
Trial registration: ANZCTR, ACTRN12624000128594. Prospectively Registered on 13 February 2024.
{"title":"LSDDEP2: study protocol for a randomised, double-dummy, triple-blind, active placebo-controlled, parallel groups trial of LSD microdosing in patients with major depressive disorder.","authors":"Dimitri Daldegan-Bueno, Carina Joy Donegan, Anna Forsyth, Rachael Louise Sumner, Robin J Murphy, David B Menkes, William Evans, Nicholas Hoeh, Frederick Sundram, Lisa M Reynolds, Rhys Ponton, Alana Cavadino, Todd Smith, Partha Roop, Nathan Allen, Binu Abeysinghe, Darren Svirskis, Mahima Bansal, Suresh Muthukumaraswamy","doi":"10.1186/s13063-024-08384-3","DOIUrl":"10.1186/s13063-024-08384-3","url":null,"abstract":"<p><strong>Background: </strong>Major depressive disorder (MDD) poses a significant global health burden with available treatments limited by inconsistent efficacy and notable side effects. Classic psychedelics, including lysergic acid diethylamide (LSD), have garnered attention for their potential in treating psychiatric disorders. Microdosing, the repeated consumption of sub-hallucinogenic doses of psychedelics, has emerged as a self-treatment approach for depression within lay communities. Building upon preliminary evidence and the successful completion of an open-label pilot trial of microdosing LSD for depression (LSDDEP1), this protocol outlines a phase 2b randomised controlled trial (LSDDEP2). The main objective of LSDDEP2 is to assess the modification of depressive symptoms, measured by the Montgomery-Åsberg Depression Rating Scale (MADRS), following a regimen of LSD microdoses versus placebo.</p><p><strong>Methods: </strong>This is a randomised, double-dummy, triple-blind, active placebo-controlled, parallel groups trial of LSD microdosing in patients meeting DSM-5 criteria for major depressive disorder. Participants will undergo an 8-week LSD microdosing regimen using the titratable MB-22001 formulation taking two doses a week. All doses will be self-administered at home and will be titratable from 4 to 20 μg based on subjective perception and tolerability. In addition to depression symptoms, outcome will include psychiatric and personality inventories, sleep and activity tracking, electroencephalography (EEG), blood biomarkers, semi-structured interviews, and safety (e.g. adverse event, laboratory exam) measures.</p><p><strong>Discussion: </strong>This study will be the first randomised controlled trial to administer controlled microdoses of LSD for treatment of MDD in participants' naturalistic environment. The measures included are designed to assess the drug's safety, mechanism, and treatment efficacy over placebo in this population. The results of this study will be important for assessing the viability of psychedelic microdosing as an additional treatment option and for informing the direction of future clinical trials.</p><p><strong>Trial registration: </strong>ANZCTR, ACTRN12624000128594. Prospectively Registered on 13 February 2024.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11344334/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056562","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: Endoscopic ultrasound (EUS)-guided transluminal drainage has become a first-line treatment modality for symptomatic pancreatic pseudocysts. Despite the increasing popularity of lumen-apposing metal stents (LAMSs), plastic stents may resolve non-necrotic fluid collections effectively with lower costs and no LAMS-specific adverse events. To date, there has been a paucity of data on the appropriate stent type in this setting. This trial aims to assess the non-inferiority of plastic stents to a LAMS for the initial EUS-guided drainage of pseudocysts.
Methods: The WONDER-02 trial is a multicentre, open-label, non-inferiority, randomised controlled trial, which will enrol pancreatic pseudocyst patients requiring EUS-guided treatment in 26 centres in Japan. This trial plans to enrol 80 patients who will be randomised at a 1:1 ratio to receive either plastic stents or a LAMS (40 patients per arm). In the plastic stent group, EUS-guided drainage will be performed using two 7-Fr double pigtail stents. In the LAMS group, the treatment will be performed in the same way except for LAMS use. The step-up treatment will be performed via endoscopic and/or percutaneous procedures at the trial investigator's discretion. The primary endpoint is clinical success, which is defined as a decrease in a pseudocyst size to ≤ 2 cm and an improvement in inflammatory indicators (i.e. body temperature, white blood cell count, and serum C-reactive protein). Secondary endpoints include technical success, adverse events including mortality, pseudocyst recurrence, and medical costs.
Discussion: The WONDER-02 trial will investigate the efficacy and safety of plastic stents compared to a LAMS in EUS-guided treatment of symptomatic pancreatic pseudocysts with a particular focus on the non-inferior efficacy of plastic stents. The findings will help establish a new treatment algorithm for this population.
Trial registration: ClinicalTrials.gov NCT06133023 registered on 9 November 2023. UMIN000052647 registered on 30 October 2023. jRCT1032230444 registered on 7 November 2023.
背景:内镜超声(EUS)引导下的腔内引流术已成为无症状胰腺假性囊肿的一线治疗方法。尽管腔隙封闭金属支架(LAMS)越来越受欢迎,但塑料支架也能有效解决非坏死性积液问题,而且成本更低,也不会出现 LAMS 特有的不良反应。迄今为止,关于在这种情况下选择何种支架类型的数据还很少。本试验旨在评估塑料支架与 LAMS 相比,在 EUS 引导下对假性囊肿进行初始引流的非劣效性:WONDER-02试验是一项多中心、开放标签、非劣效、随机对照试验,将在日本的26个中心招募需要在EUS引导下治疗的胰腺假性囊肿患者。该试验计划招募 80 名患者,按 1:1 的比例随机分配到塑料支架或 LAMS 组(每组 40 名患者)。塑料支架组将在 EUS 引导下使用两个 7 英尺双辫支架进行引流。LAMS 组除使用 LAMS 外,治疗方法相同。升级治疗将由试验研究者决定通过内窥镜和/或经皮手术进行。主要终点是临床成功,即假性囊肿缩小到≤2厘米,炎症指标(即体温、白细胞计数和血清C反应蛋白)有所改善。次要终点包括技术成功率、不良事件(包括死亡率)、假性囊肿复发和医疗费用:WONDER-02 试验将研究塑料支架与 LAMS 相比,在 EUS 引导下治疗无症状胰腺假性囊肿的疗效和安全性,尤其关注塑料支架的非劣效。研究结果将有助于为这一人群建立新的治疗算法:试验注册:ClinicalTrials.gov NCT06133023,注册日期:2023年11月9日。UMIN000052647于2023年10月30日注册。jRCT1032230444于2023年11月7日注册。
{"title":"WONDER-02: plastic stent vs. lumen-apposing metal stent for endoscopic ultrasound-guided drainage of pancreatic pseudocysts-study protocol for a multicentre randomised non-inferiority trial.","authors":"Tomotaka Saito, Mamoru Takenaka, Masaki Kuwatani, Shinpei Doi, Hiroshi Ohyama, Toshio Fujisawa, Atsuhiro Masuda, Takuji Iwashita, Hideyuki Shiomi, Nobuhiko Hayashi, Keisuke Iwata, Akinori Maruta, Tsuyoshi Mukai, Saburo Matsubara, Tsuyoshi Hamada, Tadahisa Inoue, Kazuyuki Matsumoto, Sumio Hirose, Nao Fujimori, Kosuke Kashiwabara, Hideki Kamada, Shinichi Hashimoto, Toshiyasu Shiratori, Reiko Yamada, Hirofumi Kogure, Kazunari Nakahara, Takeshi Ogura, Masayuki Kitano, Ichiro Yasuda, Hiroyuki Isayama, Yousuke Nakai","doi":"10.1186/s13063-024-08373-6","DOIUrl":"10.1186/s13063-024-08373-6","url":null,"abstract":"<p><strong>Background: </strong>Endoscopic ultrasound (EUS)-guided transluminal drainage has become a first-line treatment modality for symptomatic pancreatic pseudocysts. Despite the increasing popularity of lumen-apposing metal stents (LAMSs), plastic stents may resolve non-necrotic fluid collections effectively with lower costs and no LAMS-specific adverse events. To date, there has been a paucity of data on the appropriate stent type in this setting. This trial aims to assess the non-inferiority of plastic stents to a LAMS for the initial EUS-guided drainage of pseudocysts.</p><p><strong>Methods: </strong>The WONDER-02 trial is a multicentre, open-label, non-inferiority, randomised controlled trial, which will enrol pancreatic pseudocyst patients requiring EUS-guided treatment in 26 centres in Japan. This trial plans to enrol 80 patients who will be randomised at a 1:1 ratio to receive either plastic stents or a LAMS (40 patients per arm). In the plastic stent group, EUS-guided drainage will be performed using two 7-Fr double pigtail stents. In the LAMS group, the treatment will be performed in the same way except for LAMS use. The step-up treatment will be performed via endoscopic and/or percutaneous procedures at the trial investigator's discretion. The primary endpoint is clinical success, which is defined as a decrease in a pseudocyst size to ≤ 2 cm and an improvement in inflammatory indicators (i.e. body temperature, white blood cell count, and serum C-reactive protein). Secondary endpoints include technical success, adverse events including mortality, pseudocyst recurrence, and medical costs.</p><p><strong>Discussion: </strong>The WONDER-02 trial will investigate the efficacy and safety of plastic stents compared to a LAMS in EUS-guided treatment of symptomatic pancreatic pseudocysts with a particular focus on the non-inferior efficacy of plastic stents. The findings will help establish a new treatment algorithm for this population.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT06133023 registered on 9 November 2023. UMIN000052647 registered on 30 October 2023. jRCT1032230444 registered on 7 November 2023.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11344301/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056563","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: This randomized clinical trial protocol aimed to investigate the comparative efficacy of an enhanced recovery after surgery (ERAS) protocol versus traditional perioperative care programs in patients with intradural extramedullary spinal tumors.
Methods: The study included 180 patients aged 18-80 years, who were randomly assigned to two groups: Group A receiving traditional perioperative care and Group B receiving accelerated rehabilitation perioperative care. The nurse responsible for patient care was informed of the group assignment, but the patients themselves remained blinded to the intervention. The primary outcome measure was the Karnofsky Performance Scale score, which assessed functional status. The secondary outcomes included the Japanese Orthopedic Association Scale, Numeric Pain Rating Scale, length of postoperative hospital stay, duration of urethral catheterization, patient satisfaction questionnaire, and complication rates. Follow-up assessments were conducted telephonically 1 month, 3 months, and 6 months after the surgery.
Discussion: This study protocol provided a structured approach to assess the potential benefits of ERAS during the perioperative period for patients with intradural extramedullary tumors, aiming to improve patient outcomes and overall care efficiency.
Trial registration: This study has been registered with the China Clinical Trials Registry (Project No: ChiCTR2200063347). Registered on September 5 2022.
{"title":"Clinical evaluation of enhanced recovery versus conventional care in the perioperative period for intradural extramedullary spinal tumors: a study protocol for a multicenter, randomized controlled trial.","authors":"Jing Chen, Lishuang Chen, Xueqin Hu, Zengna Xing, Yuhui Sun, Fabin Lin, Rui Wang, Chunmei Chen, Yanjuan Lin","doi":"10.1186/s13063-024-08227-1","DOIUrl":"10.1186/s13063-024-08227-1","url":null,"abstract":"<p><strong>Background: </strong>This randomized clinical trial protocol aimed to investigate the comparative efficacy of an enhanced recovery after surgery (ERAS) protocol versus traditional perioperative care programs in patients with intradural extramedullary spinal tumors.</p><p><strong>Methods: </strong>The study included 180 patients aged 18-80 years, who were randomly assigned to two groups: Group A receiving traditional perioperative care and Group B receiving accelerated rehabilitation perioperative care. The nurse responsible for patient care was informed of the group assignment, but the patients themselves remained blinded to the intervention. The primary outcome measure was the Karnofsky Performance Scale score, which assessed functional status. The secondary outcomes included the Japanese Orthopedic Association Scale, Numeric Pain Rating Scale, length of postoperative hospital stay, duration of urethral catheterization, patient satisfaction questionnaire, and complication rates. Follow-up assessments were conducted telephonically 1 month, 3 months, and 6 months after the surgery.</p><p><strong>Discussion: </strong>This study protocol provided a structured approach to assess the potential benefits of ERAS during the perioperative period for patients with intradural extramedullary tumors, aiming to improve patient outcomes and overall care efficiency.</p><p><strong>Trial registration: </strong>This study has been registered with the China Clinical Trials Registry (Project No: ChiCTR2200063347). Registered on September 5 2022.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11344951/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-23DOI: 10.1186/s13063-024-08381-6
Mohammad Abdul Awal Miah, Jaya Chandna, Rejina Gurung, Nahya Salim Masoud, Proma Paul, Shafiqul Ameen, Omkar Basnet, Mustafa Miraji, Cally Tann, Ismat Ara Mili, A K M Tanvir Hossain, Atique Iqbal Chowdhury, Asraful Alam, Kate Mackinnon Milner, Shams El Arifeen, Ashish Kc, Karim Manji, Paul Lynch, Joy E Lawn, Jena Derakhshani Hamadani
Background: Vulnerable children, including those with neuro-developmental delays and disabilities, often face barriers in accessing early primary education, thus hindering progress toward Sustainable Development Goal 4.2. Evidence-based interventions are essential to enhancing inclusivity and establishing sustainable implementation strategies to address this challenge. This study, Every Newborn-Reach up Early Education Intervention for All Children (EN-REACH), builds on the previous Every Newborn- Simplified Measurement Integrating Longitudinal Neurodevelopmental and Growth (EN-SMILING) observational cohort study. This paper provides the protocol for a cluster randomized controlled trial (cRCT) to evaluate the effectiveness of a parenting group intervention program for enhancing school readiness in Bangladesh, Nepal, and Tanzania, and an embedded process evaluation to inform scalability and feasibility.
Methods: EN-REACH is a cRCT with at least 150 clusters to evaluate the impact of a parent training program led by trained parent-teacher facilitator pairs, focusing on children aged 4 ~ 6 years preparing for preschool. Approximately 500 participants from the EN-SMILING cohort at each site have been identified. A geographic information system will define ~ 50 clusters in each of the three countries, each with approximately ten parent-child dyads. Half the clusters will be randomly assigned to intervention and control groups. The primary outcome is "school readiness", assessed using the Measuring Early Learning Quality and Outcomes tool. Secondary outcomes include Intelligence Quotient, child functioning, growth, visual, and hearing assessments. Data will be collected at baseline, and post-intervention data following implementation of the parent group intervention sessions over approximately 5 months. Quantitative data on coverage and quality care, combined with qualitative insights from children, caregivers, facilitators, and stakeholders' perspectives, will be used to conduct a process evaluation applying the Reach, Effectiveness, Adoption, Implementation, and Maintenance (RE-AIM) framework. DISCUSSION: This protocol details a trial focused on enhancing school readiness and cognitive abilities in young children, inclusive of those with disabilities, aiming to bridge gap from home to early primary education. EN-REACH aims to provide insights into the effectiveness and acceptability of a co-designed disability-inclusive school readiness program in three countries, potentially impacting national and global policies for all children, including those with disabilities.
Trial registration: The trial was retrospectively registered on clinicaltrials.gov on 29 February 2024 (NCT06334627).
{"title":"Every Newborn-Reach Up Early Education Intervention for All Children (EN-REACH)- a parent group intervention for school readiness in Bangladesh, Nepal, and Tanzania: study protocol for a cluster randomized controlled trial.","authors":"Mohammad Abdul Awal Miah, Jaya Chandna, Rejina Gurung, Nahya Salim Masoud, Proma Paul, Shafiqul Ameen, Omkar Basnet, Mustafa Miraji, Cally Tann, Ismat Ara Mili, A K M Tanvir Hossain, Atique Iqbal Chowdhury, Asraful Alam, Kate Mackinnon Milner, Shams El Arifeen, Ashish Kc, Karim Manji, Paul Lynch, Joy E Lawn, Jena Derakhshani Hamadani","doi":"10.1186/s13063-024-08381-6","DOIUrl":"10.1186/s13063-024-08381-6","url":null,"abstract":"<p><strong>Background: </strong>Vulnerable children, including those with neuro-developmental delays and disabilities, often face barriers in accessing early primary education, thus hindering progress toward Sustainable Development Goal 4.2. Evidence-based interventions are essential to enhancing inclusivity and establishing sustainable implementation strategies to address this challenge. This study, Every Newborn-Reach up Early Education Intervention for All Children (EN-REACH), builds on the previous Every Newborn- Simplified Measurement Integrating Longitudinal Neurodevelopmental and Growth (EN-SMILING) observational cohort study. This paper provides the protocol for a cluster randomized controlled trial (cRCT) to evaluate the effectiveness of a parenting group intervention program for enhancing school readiness in Bangladesh, Nepal, and Tanzania, and an embedded process evaluation to inform scalability and feasibility.</p><p><strong>Methods: </strong>EN-REACH is a cRCT with at least 150 clusters to evaluate the impact of a parent training program led by trained parent-teacher facilitator pairs, focusing on children aged 4 ~ 6 years preparing for preschool. Approximately 500 participants from the EN-SMILING cohort at each site have been identified. A geographic information system will define ~ 50 clusters in each of the three countries, each with approximately ten parent-child dyads. Half the clusters will be randomly assigned to intervention and control groups. The primary outcome is \"school readiness\", assessed using the Measuring Early Learning Quality and Outcomes tool. Secondary outcomes include Intelligence Quotient, child functioning, growth, visual, and hearing assessments. Data will be collected at baseline, and post-intervention data following implementation of the parent group intervention sessions over approximately 5 months. Quantitative data on coverage and quality care, combined with qualitative insights from children, caregivers, facilitators, and stakeholders' perspectives, will be used to conduct a process evaluation applying the Reach, Effectiveness, Adoption, Implementation, and Maintenance (RE-AIM) framework. DISCUSSION: This protocol details a trial focused on enhancing school readiness and cognitive abilities in young children, inclusive of those with disabilities, aiming to bridge gap from home to early primary education. EN-REACH aims to provide insights into the effectiveness and acceptability of a co-designed disability-inclusive school readiness program in three countries, potentially impacting national and global policies for all children, including those with disabilities.</p><p><strong>Trial registration: </strong>The trial was retrospectively registered on clinicaltrials.gov on 29 February 2024 (NCT06334627).</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11342614/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142047260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-23DOI: 10.1186/s13063-024-08380-7
Rachel Deere, Philip Pallmann, Victoria Shepherd, Lucy Brookes-Howell, Andrew Carson-Stevens, Ffion Davies, Emma Dunphy, Preeti Gupta, Mary Hickson, Val Hill, Kate Ingarfield, Nicola Ivins, Fiona Jones, Robert Letchford, Rachel Lowe, Sarah Nash, Paula Otter, Hayley Prout, Elizabeth Randell, Bernadette Sewell, Debs Smith, Robert Trubey, Tom Wainwright, Monica Busse, Kate Button
Background: Four out of five people living with osteoarthritis (OA) also suffer with at least one other long-term health condition. The complex interaction between OA and multiple long-term conditions (MLTCs) can result in difficulties with self-care, restricted mobility, pain, anxiety, depression and reduced quality of life. The aim of the MulTI-domain Self-management in Older People wiTh OstEoarthritis and Multi-Morbidities (TIPTOE) trial is to evaluate the clinical and cost-effectiveness of the Living Well self-management support intervention, co-designed with people living with OA, integrated into usual care, in comparison to usual care alone.
Methods: TIPTOE is a multi-centre, two-arm, individually randomised controlled trial where 824 individuals over 65 years old with knee and/or hip joint pain from their OA affected joint and at least one other long-term health condition will be randomised to receive either the Living Well Self-Management support intervention or usual care. Eligible participants can self-refer onto the trial via a website or be referred via NHS services across Wales and England. Those randomised to receive the Living Well support intervention will be offered up to six one-to-one coaching sessions with a TIPTOE-trained healthcare practitioner and a co-designed book. Participants will be encouraged to nominate a support person to assist them throughout the study. All participants will complete a series of self-reported outcome measures at baseline and 6- and 12-month follow-up. The primary outcome is symptoms and quality of life as assessed by the Musculoskeletal Health Questionnaire (MSK-HQ). Routine data will be used to evaluate health resource use. A mixed methods process evaluation will be conducted alongside the trial to inform future implementation should the TIPTOE intervention be found both clinically and cost-effective. An embedded 'Study Within A Project' (SWAP) will explore and address barriers to the inclusion of under-served patient groups (e.g. oldest old, low socioeconomic groups, ethnic groups).
Discussion: TIPTOE will evaluate the clinical and cost-effectiveness of a co-designed, living well personalised self-management support intervention for older individuals with knee and/or hip OA and MLTCs. The trial has been designed to maximise inclusivity and access.
Trial registration: ISRCTN 16024745 . Registered on October 16, 2023.
{"title":"MulTI-domain self-management in older People wiTh OstEoarthritis and multi-morbidities: protocol for the TIPTOE randomised controlled trial.","authors":"Rachel Deere, Philip Pallmann, Victoria Shepherd, Lucy Brookes-Howell, Andrew Carson-Stevens, Ffion Davies, Emma Dunphy, Preeti Gupta, Mary Hickson, Val Hill, Kate Ingarfield, Nicola Ivins, Fiona Jones, Robert Letchford, Rachel Lowe, Sarah Nash, Paula Otter, Hayley Prout, Elizabeth Randell, Bernadette Sewell, Debs Smith, Robert Trubey, Tom Wainwright, Monica Busse, Kate Button","doi":"10.1186/s13063-024-08380-7","DOIUrl":"10.1186/s13063-024-08380-7","url":null,"abstract":"<p><strong>Background: </strong>Four out of five people living with osteoarthritis (OA) also suffer with at least one other long-term health condition. The complex interaction between OA and multiple long-term conditions (MLTCs) can result in difficulties with self-care, restricted mobility, pain, anxiety, depression and reduced quality of life. The aim of the MulTI-domain Self-management in Older People wiTh OstEoarthritis and Multi-Morbidities (TIPTOE) trial is to evaluate the clinical and cost-effectiveness of the Living Well self-management support intervention, co-designed with people living with OA, integrated into usual care, in comparison to usual care alone.</p><p><strong>Methods: </strong>TIPTOE is a multi-centre, two-arm, individually randomised controlled trial where 824 individuals over 65 years old with knee and/or hip joint pain from their OA affected joint and at least one other long-term health condition will be randomised to receive either the Living Well Self-Management support intervention or usual care. Eligible participants can self-refer onto the trial via a website or be referred via NHS services across Wales and England. Those randomised to receive the Living Well support intervention will be offered up to six one-to-one coaching sessions with a TIPTOE-trained healthcare practitioner and a co-designed book. Participants will be encouraged to nominate a support person to assist them throughout the study. All participants will complete a series of self-reported outcome measures at baseline and 6- and 12-month follow-up. The primary outcome is symptoms and quality of life as assessed by the Musculoskeletal Health Questionnaire (MSK-HQ). Routine data will be used to evaluate health resource use. A mixed methods process evaluation will be conducted alongside the trial to inform future implementation should the TIPTOE intervention be found both clinically and cost-effective. An embedded 'Study Within A Project' (SWAP) will explore and address barriers to the inclusion of under-served patient groups (e.g. oldest old, low socioeconomic groups, ethnic groups).</p><p><strong>Discussion: </strong>TIPTOE will evaluate the clinical and cost-effectiveness of a co-designed, living well personalised self-management support intervention for older individuals with knee and/or hip OA and MLTCs. The trial has been designed to maximise inclusivity and access.</p><p><strong>Trial registration: </strong>ISRCTN 16024745 . Registered on October 16, 2023.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11344358/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142047261","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: Tracheal intubation may cause significant hemodynamic responses. Many drugs have been shown to be effective in modifying these cardiovascular responses, including remifentanil, fentanyl, sufentanil, and alfentanil. However, the 90% effect-site concentration (EC90) of remifentanil required to control cardiovascular responses to tracheal intubation when combined with ciprofol remains unclear. The purpose of this study was to determine the EC90 of remifentanil inhibiting cardiovascular responses to tracheal intubation during anesthesia induction with ciprofol using biased-coin design up-and-down sequential method (BC-UDM).
Methods: This is a prospective sequential allocation dose-finding study. American Society of Anesthesiologists physical status (ASA) I-II elective surgical patients receiving target-controlled infusion (TCI) of remifentanil effect-site concentration (Ce), followed by ciprofol and rocuronium for anesthesia, were enrolled. The cardiovascular response to tracheal intubation was defined as positive when mean arterial pressure (MAP) or heart rate (HR) is 15% higher than the baseline value. Using the BC-UDM, the Ce of remifentanil was determined based on the cardiovascular response to tracheal intubation of the previous patient. The EC90 and 90% confidence intervals (90% CIs) were estimated by R-Foundation centered isotonic regression and the pooled adjacent violators algorithm with bootstrapping.
Discussion: The results of this study sought to demonstrate EC90 of remifentanil blunting sympathetic responses to tracheal intubation during anesthesia index (Ai)-guided ciprofol anesthesia using BCD-UDM. It may help to minimize the cardiovascular responses to tracheal intubation.
Trial registration: Chinese Clinical Trial Registry ChiCTR2300078275. Registered on December 3, 2023.
{"title":"The EC90 of remifentanil for inhibiting endotracheal intubation responses under anesthesia induction with ciprofol: study protocol for a dose-finding trial with the biased-coin design.","authors":"Jianing Guo, Fangsheng Xu, Luoyun Li, Zeru Zhang, Baichun Xing, Qin Fan, Zehua Wang, Chunyu Li","doi":"10.1186/s13063-024-08397-y","DOIUrl":"10.1186/s13063-024-08397-y","url":null,"abstract":"<p><strong>Background: </strong>Tracheal intubation may cause significant hemodynamic responses. Many drugs have been shown to be effective in modifying these cardiovascular responses, including remifentanil, fentanyl, sufentanil, and alfentanil. However, the 90% effect-site concentration (EC90) of remifentanil required to control cardiovascular responses to tracheal intubation when combined with ciprofol remains unclear. The purpose of this study was to determine the EC90 of remifentanil inhibiting cardiovascular responses to tracheal intubation during anesthesia induction with ciprofol using biased-coin design up-and-down sequential method (BC-UDM).</p><p><strong>Methods: </strong>This is a prospective sequential allocation dose-finding study. American Society of Anesthesiologists physical status (ASA) I-II elective surgical patients receiving target-controlled infusion (TCI) of remifentanil effect-site concentration (Ce), followed by ciprofol and rocuronium for anesthesia, were enrolled. The cardiovascular response to tracheal intubation was defined as positive when mean arterial pressure (MAP) or heart rate (HR) is 15% higher than the baseline value. Using the BC-UDM, the Ce of remifentanil was determined based on the cardiovascular response to tracheal intubation of the previous patient. The EC90 and 90% confidence intervals (90% CIs) were estimated by R-Foundation centered isotonic regression and the pooled adjacent violators algorithm with bootstrapping.</p><p><strong>Discussion: </strong>The results of this study sought to demonstrate EC90 of remifentanil blunting sympathetic responses to tracheal intubation during anesthesia index (Ai)-guided ciprofol anesthesia using BCD-UDM. It may help to minimize the cardiovascular responses to tracheal intubation.</p><p><strong>Trial registration: </strong>Chinese Clinical Trial Registry ChiCTR2300078275. Registered on December 3, 2023.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11344379/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142047262","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-22DOI: 10.1186/s13063-024-08378-1
Esther Nakyaze, Suzanne Van Hulle, John Hembling, Emmanuel Arinaitwe, Momar Mbodji, Mary Grace Alwano, Felly Christine Lamwaka, Stephen Tukwasibwe, Samuel Gonahasa, Fang Liu, John P Grieco, Nicole L Achee
Background: Spatial repellents (SRs) have been widely used for the prevention of mosquito bites, and preliminary findings suggest efficacy against both malaria (1) and Aedes-borne viruses (2) but their effectiveness in reducing mosquito-borne diseases under operational use has never been evaluated. SRs have the potential of being critical tools in the prevention of mosquito-borne diseases in contexts where typical vector control strategies, such as insecticide-treated nets (ITNs) and indoor residual spraying, are inaccessible or underutilized such as among displaced persons or in emergency relief settings.
Methods: Children will be enrolled in 3 separate cohorts to establish the effectiveness of SRs in reducing malaria infection in different distribution channels. One cohort will estimate the direct effect of the SR distributed through a reference channel (study personnel distribution). The two remaining cohorts will estimate the protection of the SR distributed through a voucher channel and the Village Health Team channel. Cohorts will be followed twice a month (approximately every 15 days): during the first scheduled household visit in the month, a blood sample will be taken for malaria rapid diagnostic test (Monthly Visit #1); and, during the second scheduled household visit, a blood sample will only be taken if the participant has a recent history of fever (Monthly Visit #2). The incidence of malaria in each cohort will be estimated and compared to the reference cohort to determine the benefit of using a SR in an area with high, year-round transmission of malaria.
Discussion: This study will address the knowledge gap of whether or not SRs are effective in reducing human malaria disease in humanitarian assistance and emergency response settings in sub-Saharan Africa where underlying transmission rates are historically high and ITNs may or may not be widely deployed. This research will inform policy makers on whether to recommend SRs as a means to further reduce malaria transmission for such operational programs.
Trial registration: ClinicalTrials.gov NCT06122142. Registered on November 8, 2023.
{"title":"Advancing spatial repellents for malaria control: effectiveness and cost-effectiveness of a spatial repellent under operational use in Northern Uganda-study protocol for a cluster randomized controlled trial.","authors":"Esther Nakyaze, Suzanne Van Hulle, John Hembling, Emmanuel Arinaitwe, Momar Mbodji, Mary Grace Alwano, Felly Christine Lamwaka, Stephen Tukwasibwe, Samuel Gonahasa, Fang Liu, John P Grieco, Nicole L Achee","doi":"10.1186/s13063-024-08378-1","DOIUrl":"10.1186/s13063-024-08378-1","url":null,"abstract":"<p><strong>Background: </strong>Spatial repellents (SRs) have been widely used for the prevention of mosquito bites, and preliminary findings suggest efficacy against both malaria (1) and Aedes-borne viruses (2) but their effectiveness in reducing mosquito-borne diseases under operational use has never been evaluated. SRs have the potential of being critical tools in the prevention of mosquito-borne diseases in contexts where typical vector control strategies, such as insecticide-treated nets (ITNs) and indoor residual spraying, are inaccessible or underutilized such as among displaced persons or in emergency relief settings.</p><p><strong>Methods: </strong>Children will be enrolled in 3 separate cohorts to establish the effectiveness of SRs in reducing malaria infection in different distribution channels. One cohort will estimate the direct effect of the SR distributed through a reference channel (study personnel distribution). The two remaining cohorts will estimate the protection of the SR distributed through a voucher channel and the Village Health Team channel. Cohorts will be followed twice a month (approximately every 15 days): during the first scheduled household visit in the month, a blood sample will be taken for malaria rapid diagnostic test (Monthly Visit #1); and, during the second scheduled household visit, a blood sample will only be taken if the participant has a recent history of fever (Monthly Visit #2). The incidence of malaria in each cohort will be estimated and compared to the reference cohort to determine the benefit of using a SR in an area with high, year-round transmission of malaria.</p><p><strong>Discussion: </strong>This study will address the knowledge gap of whether or not SRs are effective in reducing human malaria disease in humanitarian assistance and emergency response settings in sub-Saharan Africa where underlying transmission rates are historically high and ITNs may or may not be widely deployed. This research will inform policy makers on whether to recommend SRs as a means to further reduce malaria transmission for such operational programs.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT06122142. Registered on November 8, 2023.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11342661/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142037134","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-22DOI: 10.1186/s13063-024-08393-2
Muram El-Nayir, Rohan Wijesurendra, David Preiss, Marion Mafham, Leandros Tsiotos, Sadman Islam, Anne Whitehouse, Sophia Wilkinson, Hannah Freeman, Ryonfa Lee, Wojciech Brudlo, Genna Bobby, Bryony Jenkins, Robert Humphrey, Amy Mallorie, Andrew Toal, Elnora C Barker, Dianna Moylan, Graeme Thomson, Firoza Davies, Hameed Khan, Ian Allotey, Susan Dickie, John Roberts
Introduction: ASCEND PLUS is a randomised controlled trial assessing the effects of oral semaglutide on the primary prevention of cardiovascular events in around 20,000 individuals with type 2 diabetes in the UK. The trial's innovative design includes a decentralised direct-to-participant invitation, recruitment, and follow-up model, relying on self-completion of online forms or telephone or video calls with research nurses, with no physical sites. Extensive patient and public involvement and engagement (PPIE) was essential to the design and conduct of ASCEND PLUS.
Aim: To report the process and conduct of PPIE activity in ASCEND PLUS, evaluate effects on trial design, reflect critically on successes and aspects that could have been improved, and identify themes and learning relevant to implementation of PPIE in future trials.
Methods: PPIE activity was coordinated centrally and included six PPIE focus groups and creation of an ASCEND PLUS public advisory group (PAG) during the design phase. Recruitment to these groups was carefully considered to ensure diversity and inclusion, largely consisting of adults living with type 2 diabetes from across the UK. Two members of the PAG also joined the trial Steering Committee. Steering Committee meetings, focus groups, and PAG meetings were conducted online, with two hybrid workshops to discuss PPIE activity and aspects of the trial.
Results: PPIE activity was critical to shaping the design and conduct of ASCEND PLUS. Key examples included supporting choice for participants to either complete the screening/consent process independently online, or during a telephone or video call interview with a research nurse. A concise 'initial information leaflet' was developed to be sent with the initial invitations, with the 'full' information leaflet sent later to those interested in joining the trial. The PAG reviewed the content and format of participant- and public-facing materials, including written documents, online screening forms, animated videos, and the trial website, to aid clarity and accessibility, and provided input into the choice of instruments to assess quality of life.
Conclusions: PPIE is integral in ASCEND PLUS and will continue throughout the trial. This involvement has been critical to optimising the trial design, successfully obtaining regulatory and ethical approval, and conducting the trial.
简介ASCEND PLUS 是一项随机对照试验,旨在评估口服塞马鲁肽对英国约 2 万名 2 型糖尿病患者心血管事件一级预防的效果。该试验的创新设计包括一种分散的直接面向参与者的邀请、招募和随访模式,依赖于在线表格的自我填写或与研究护士的电话或视频通话,没有实体试验点。广泛的患者和公众参与(PPIE)对ASCEND PLUS的设计和实施至关重要。目的:报告ASCEND PLUS中PPIE活动的过程和实施情况,评估对试验设计的影响,批判性地反思成功之处和可以改进的方面,并确定在未来试验中实施PPIE的相关主题和经验:PPIE活动由中央协调,包括六个PPIE焦点小组,并在设计阶段成立了ASCEND PLUS公众咨询小组(PAG)。这些小组的招募工作经过仔细考虑,以确保多样性和包容性,主要由英国各地的 2 型糖尿病成人患者组成。公众咨询小组的两名成员还加入了试验指导委员会。指导委员会会议、焦点小组会议和 PAG 会议均在网上举行,同时还举行了两次混合研讨会,讨论 PPIE 活动和试验的各个方面:PPIE 活动对 ASCEND PLUS 的设计和实施至关重要。主要例子包括支持参与者选择在线独立完成筛查/同意程序,或与研究护士进行电话或视频通话访谈。我们制作了一份简明的 "初始信息传单",随初始邀请函一起寄出,"完整 "信息传单稍后会寄给有兴趣参加试验的人。PAG 审查了面向参与者和公众的材料的内容和格式,包括书面文件、在线筛查表、动画视频和试验网站,以提高清晰度和可访问性,并为生活质量评估工具的选择提供意见:PPIE 是 ASCEND PLUS 试验不可或缺的一部分,并将贯穿整个试验过程。这种参与对于优化试验设计、成功获得监管和伦理批准以及开展试验至关重要。
{"title":"Patient and public involvement and engagement in the ASCEND PLUS trial: reflections from the design of a streamlined and decentralised clinical trial.","authors":"Muram El-Nayir, Rohan Wijesurendra, David Preiss, Marion Mafham, Leandros Tsiotos, Sadman Islam, Anne Whitehouse, Sophia Wilkinson, Hannah Freeman, Ryonfa Lee, Wojciech Brudlo, Genna Bobby, Bryony Jenkins, Robert Humphrey, Amy Mallorie, Andrew Toal, Elnora C Barker, Dianna Moylan, Graeme Thomson, Firoza Davies, Hameed Khan, Ian Allotey, Susan Dickie, John Roberts","doi":"10.1186/s13063-024-08393-2","DOIUrl":"10.1186/s13063-024-08393-2","url":null,"abstract":"<p><strong>Introduction: </strong>ASCEND PLUS is a randomised controlled trial assessing the effects of oral semaglutide on the primary prevention of cardiovascular events in around 20,000 individuals with type 2 diabetes in the UK. The trial's innovative design includes a decentralised direct-to-participant invitation, recruitment, and follow-up model, relying on self-completion of online forms or telephone or video calls with research nurses, with no physical sites. Extensive patient and public involvement and engagement (PPIE) was essential to the design and conduct of ASCEND PLUS.</p><p><strong>Aim: </strong>To report the process and conduct of PPIE activity in ASCEND PLUS, evaluate effects on trial design, reflect critically on successes and aspects that could have been improved, and identify themes and learning relevant to implementation of PPIE in future trials.</p><p><strong>Methods: </strong>PPIE activity was coordinated centrally and included six PPIE focus groups and creation of an ASCEND PLUS public advisory group (PAG) during the design phase. Recruitment to these groups was carefully considered to ensure diversity and inclusion, largely consisting of adults living with type 2 diabetes from across the UK. Two members of the PAG also joined the trial Steering Committee. Steering Committee meetings, focus groups, and PAG meetings were conducted online, with two hybrid workshops to discuss PPIE activity and aspects of the trial.</p><p><strong>Results: </strong>PPIE activity was critical to shaping the design and conduct of ASCEND PLUS. Key examples included supporting choice for participants to either complete the screening/consent process independently online, or during a telephone or video call interview with a research nurse. A concise 'initial information leaflet' was developed to be sent with the initial invitations, with the 'full' information leaflet sent later to those interested in joining the trial. The PAG reviewed the content and format of participant- and public-facing materials, including written documents, online screening forms, animated videos, and the trial website, to aid clarity and accessibility, and provided input into the choice of instruments to assess quality of life.</p><p><strong>Conclusions: </strong>PPIE is integral in ASCEND PLUS and will continue throughout the trial. This involvement has been critical to optimising the trial design, successfully obtaining regulatory and ethical approval, and conducting the trial.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11340079/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142037135","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-22DOI: 10.1186/s13063-024-08399-w
Elin M Swärd, Jonas Beckman, Farnoush Tabaroj, Maria K Wilcke
Background: Osteoarthritis (OA) contributes increasingly to disability worldwide. There is ample high-quality research on the treatment of knee and hip OA, whereas research on surgical and non-surgical treatment in hand OA is sparse. Limited evidence suggests that education and exercise may improve pain, function, stiffness, and grip strength in hand OA. The established surgical options in hand OA have disadvantages. Prostheses preserve motion but have a high complication rate, whereas fusions decrease function due to limited movement. There is an unmet need for high-quality research on treatment options for hand OA and a need for the development of effective and safe movement-sparing therapies. This study aims to compare the effects of a motion-preserving surgical treatment (denervation of the proximal interphalangeal (PIP) joint) with a patient education and exercise program on patient-reported outcomes and objective function in painful PIP OA.
Methods: In this parallel-group, two-armed, randomized, controlled superiority trial (RCT), 90 participants are assigned to surgical PIP joint denervation or education and exercise. Pain on load 1 year after intervention is the primary outcome measure. Secondary outcome measures include pain at rest, Patient-Rated Wrist and Hand Evaluation (PRWHE), HQ8 score, EQ5D-5L, objective physical function, complications, two-point discrimination, Mini Sollerman, consumption of analgesics, and the need for further surgery. Assessments are performed at baseline, 3 and 6 months, and 1 year after intervention.
Discussion: There are no previous RCTs comparing surgical and non-surgical treatment in PIP OA. If patient education plus exercise or PIP denervation improve function, these treatments could be implemented as first-line treatment options in PIP OA. However, if denervation does not achieve better results than non-surgical treatment, it is not justified to use in PIP OA.
Trial registration: Prospectively registered in ClinicalTrials.gov (NCT05980793) on 8 August 2023. URL https://classic.
Clinicaltrials: gov/ct2/show/NCT05980793 .
背景:骨关节炎(OA)越来越多地导致全球范围内的残疾。有关膝关节和髋关节 OA 治疗的高质量研究很多,但有关手部 OA 手术和非手术治疗的研究却很少。有限的证据表明,教育和锻炼可以改善手部OA的疼痛、功能、僵硬和握力。治疗手部 OA 的既有手术方案也有缺点。假肢可以保持活动,但并发症发生率较高,而融合术则会因活动受限而降低功能。对手部 OA 治疗方案进行高质量研究的需求尚未得到满足,因此需要开发有效、安全的保护运动疗法。本研究旨在比较保留运动的手术治疗(近端指间关节(PIP)神经支配)与患者教育和锻炼计划对疼痛性 PIP OA 患者报告结果和客观功能的影响:在这项平行分组、双臂随机对照优效试验(RCT)中,90名参与者被分配接受PIP关节去神经化手术或教育和锻炼。干预1年后负重时的疼痛是主要的结果测量指标。次要结果指标包括静息时的疼痛、患者评定的腕部和手部评估(PRWHE)、HQ8 评分、EQ5D-5L、客观身体功能、并发症、两点辨别力、Mini Sollerman、止痛药消耗量以及是否需要进一步手术。评估在基线、3个月和6个月以及干预后1年进行:讨论:目前还没有任何一项研究对PIP OA的手术治疗和非手术治疗进行比较。如果患者教育加锻炼或腓骨神经支配能改善患者的功能,那么这些治疗方法可作为腓骨骨性关节炎的一线治疗方案。但是,如果去神经化治疗不能取得比非手术治疗更好的效果,那么就没有理由将其用于PIP OA:试验注册:2023年8月8日在ClinicalTrials.gov(NCT05980793)进行了前瞻性注册。URL https://classic.Clinicaltrials: gov/ct2/show/NCT05980793 。
{"title":"Efficacy of denervation for osteoarthritis in the proximal interphalangeal joint (DOP): protocol of a randomized controlled trial.","authors":"Elin M Swärd, Jonas Beckman, Farnoush Tabaroj, Maria K Wilcke","doi":"10.1186/s13063-024-08399-w","DOIUrl":"10.1186/s13063-024-08399-w","url":null,"abstract":"<p><strong>Background: </strong>Osteoarthritis (OA) contributes increasingly to disability worldwide. There is ample high-quality research on the treatment of knee and hip OA, whereas research on surgical and non-surgical treatment in hand OA is sparse. Limited evidence suggests that education and exercise may improve pain, function, stiffness, and grip strength in hand OA. The established surgical options in hand OA have disadvantages. Prostheses preserve motion but have a high complication rate, whereas fusions decrease function due to limited movement. There is an unmet need for high-quality research on treatment options for hand OA and a need for the development of effective and safe movement-sparing therapies. This study aims to compare the effects of a motion-preserving surgical treatment (denervation of the proximal interphalangeal (PIP) joint) with a patient education and exercise program on patient-reported outcomes and objective function in painful PIP OA.</p><p><strong>Methods: </strong>In this parallel-group, two-armed, randomized, controlled superiority trial (RCT), 90 participants are assigned to surgical PIP joint denervation or education and exercise. Pain on load 1 year after intervention is the primary outcome measure. Secondary outcome measures include pain at rest, Patient-Rated Wrist and Hand Evaluation (PRWHE), HQ8 score, EQ5D-5L, objective physical function, complications, two-point discrimination, Mini Sollerman, consumption of analgesics, and the need for further surgery. Assessments are performed at baseline, 3 and 6 months, and 1 year after intervention.</p><p><strong>Discussion: </strong>There are no previous RCTs comparing surgical and non-surgical treatment in PIP OA. If patient education plus exercise or PIP denervation improve function, these treatments could be implemented as first-line treatment options in PIP OA. However, if denervation does not achieve better results than non-surgical treatment, it is not justified to use in PIP OA.</p><p><strong>Trial registration: </strong>Prospectively registered in ClinicalTrials.gov (NCT05980793) on 8 August 2023. URL https://classic.</p><p><strong>Clinicaltrials: </strong>gov/ct2/show/NCT05980793 .</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11340183/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142018775","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-21DOI: 10.1186/s13063-024-08382-5
Hao T M Bui, Le Minh Giang, Jane S Chen, Teerada Sripaipan, Ha T T Nong, Ngan T K Nguyen, Sophia M Bartels, Sarah L Rossi, Heidi Hutton, Geetanjali Chander, Hojoon Sohn, Olivia Ferguson, Ha V Tran, Minh X Nguyen, Khanh D Nguyen, Sarah E Rutstein, Sara Levintow, Irving F Hoffman, Byron J Powell, Brian W Pence, Vivian F Go, William C Miller
Background: In Vietnam and other global settings, men who have sex with men (MSM) have become the population at greatest risk of HIV infection. Although HIV pre-exposure prophylaxis (PrEP) has been implemented as a prevention strategy, PrEP outcomes may be affected by low persistence and adherence among MSM with unhealthy alcohol use. MSM have a high prevalence of unhealthy alcohol use in Vietnam, which may affect PrEP outcomes.
Methods: Design: We will conduct a two-arm hybrid type 1 effectiveness-implementation randomized controlled trial of a brief alcohol intervention (BAI) compared to the standard of care (SOC) at the Sexual Health Promotion (SHP) clinic Hanoi, Vietnam.
Participants: Sexually active MSM (n=564) who are newly initiating PrEP or re-initiating PrEP and have unhealthy alcohol use will be recruited and randomized 1:1 to the SOC or BAI arm. A subgroup of participants (n=20) in each arm will be selected for longitudinal qualitative interviews; an additional subset (n=48) in the BAI arm will complete brief quantitative and qualitative interviews after completion of the BAI to assess the acceptability of the intervention. Additional implementation outcomes will be assessed through interviews with clinic staff and stakeholders (n=35).
Intervention: Study participants in both arms will receive standard care for PrEP clients. In the BAI arm, each participant will receive two face-to-face intervention sessions and two brief booster phone sessions, based on cognitive behavioral therapy and delivered in motivational interviewing informed style, to address their unhealthy alcohol use.
Outcomes: Effectiveness (PrEP and alcohol use) and cost-effectiveness outcomes will be compared between the two arms. Intervention implementation outcomes (acceptability, feasibility, adoption) will be assessed among MSM participants, clinic staff, and stakeholders.
Discussion: This proposed trial will assess an alcohol intervention for MSM with unhealthy alcohol use who initiate or re-initiate PrEP, while simultaneously preparing for subsequent implementation. The study will measure the effectiveness of the BAI for increasing PrEP persistence through reducing unhealthy alcohol use in a setting where excessive alcohol consumption is a normative behavior. If effective, implementation-focused results will inform future scale-up of the BAI in similar settings.
Trial registration: NCT06094634 on clinicaltrials.gov. Registered 16 October 2023.
背景:在越南和全球其他地区,男男性行为者(MSM)已成为艾滋病感染风险最大的人群。虽然艾滋病暴露前预防(PrEP)已被作为一种预防策略来实施,但PrEP的效果可能会受到酗酒不健康的男男性行为者坚持性和依从性低的影响。在越南,男男性行为者不健康饮酒的发生率很高,这可能会影响 PrEP 的效果:方法:设计:我们将在越南河内的性健康促进(SHP)诊所开展一项两臂混合型 1 类有效性实施随机对照试验,将简短酒精干预(BAI)与标准护理(SOC)进行比较:性活跃的 MSM(人数=564)将被招募,他们新近开始或重新开始 PrEP,并且饮酒不健康,他们将被 1:1 随机分配到 SOC 或 BAI 组。将在每组参与者中选取一个子组(n=20)进行纵向定性访谈;BAI 组中的另一个子组(n=48)将在 BAI 完成后完成简短的定量和定性访谈,以评估干预措施的可接受性。其他实施结果将通过对诊所工作人员和利益相关者(人数=35)的访谈进行评估:干预措施:两组研究参与者都将接受 PrEP 客户的标准护理。在 BAI 部分,每位参与者将接受两次面对面的干预治疗和两次简短的强化电话治疗,这些治疗以认知行为疗法为基础,以动机访谈的方式进行,以解决他们不健康的饮酒问题:结果:将对两组干预的效果(PrEP 和饮酒)和成本效益进行比较。干预措施的实施结果(可接受性、可行性、采用率)将在 MSM 参与者、诊所工作人员和利益相关者中进行评估:这项拟议的试验将评估针对开始或重新开始 PrEP 的不健康饮酒 MSM 的酒精干预措施,同时为后续实施做好准备。在过度饮酒是一种规范行为的环境中,该研究将衡量 BAI 通过减少不健康饮酒来提高 PrEP 持续性的效果。如果有效,以实施为重点的结果将为今后在类似环境中推广 BAI 提供参考:试验注册:Clinicaltrials.gov 上的 NCT06094634。注册日期:2023 年 10 月 16 日。
{"title":"A Brief Alcohol Intervention (BAI) to reduce alcohol use and improve PrEP outcomes among men who have sex with men in Vietnam: study protocol for a randomized controlled trial.","authors":"Hao T M Bui, Le Minh Giang, Jane S Chen, Teerada Sripaipan, Ha T T Nong, Ngan T K Nguyen, Sophia M Bartels, Sarah L Rossi, Heidi Hutton, Geetanjali Chander, Hojoon Sohn, Olivia Ferguson, Ha V Tran, Minh X Nguyen, Khanh D Nguyen, Sarah E Rutstein, Sara Levintow, Irving F Hoffman, Byron J Powell, Brian W Pence, Vivian F Go, William C Miller","doi":"10.1186/s13063-024-08382-5","DOIUrl":"10.1186/s13063-024-08382-5","url":null,"abstract":"<p><strong>Background: </strong>In Vietnam and other global settings, men who have sex with men (MSM) have become the population at greatest risk of HIV infection. Although HIV pre-exposure prophylaxis (PrEP) has been implemented as a prevention strategy, PrEP outcomes may be affected by low persistence and adherence among MSM with unhealthy alcohol use. MSM have a high prevalence of unhealthy alcohol use in Vietnam, which may affect PrEP outcomes.</p><p><strong>Methods: </strong>Design: We will conduct a two-arm hybrid type 1 effectiveness-implementation randomized controlled trial of a brief alcohol intervention (BAI) compared to the standard of care (SOC) at the Sexual Health Promotion (SHP) clinic Hanoi, Vietnam.</p><p><strong>Participants: </strong>Sexually active MSM (n=564) who are newly initiating PrEP or re-initiating PrEP and have unhealthy alcohol use will be recruited and randomized 1:1 to the SOC or BAI arm. A subgroup of participants (n=20) in each arm will be selected for longitudinal qualitative interviews; an additional subset (n=48) in the BAI arm will complete brief quantitative and qualitative interviews after completion of the BAI to assess the acceptability of the intervention. Additional implementation outcomes will be assessed through interviews with clinic staff and stakeholders (n=35).</p><p><strong>Intervention: </strong>Study participants in both arms will receive standard care for PrEP clients. In the BAI arm, each participant will receive two face-to-face intervention sessions and two brief booster phone sessions, based on cognitive behavioral therapy and delivered in motivational interviewing informed style, to address their unhealthy alcohol use.</p><p><strong>Outcomes: </strong>Effectiveness (PrEP and alcohol use) and cost-effectiveness outcomes will be compared between the two arms. Intervention implementation outcomes (acceptability, feasibility, adoption) will be assessed among MSM participants, clinic staff, and stakeholders.</p><p><strong>Discussion: </strong>This proposed trial will assess an alcohol intervention for MSM with unhealthy alcohol use who initiate or re-initiate PrEP, while simultaneously preparing for subsequent implementation. The study will measure the effectiveness of the BAI for increasing PrEP persistence through reducing unhealthy alcohol use in a setting where excessive alcohol consumption is a normative behavior. If effective, implementation-focused results will inform future scale-up of the BAI in similar settings.</p><p><strong>Trial registration: </strong>NCT06094634 on clinicaltrials.gov. Registered 16 October 2023.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11337901/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142009522","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}