Contemporary Clinical Trials Communications最新文献

Introduction

Children with Cerebral Palsy (CP) encounter substantial nutritional challenges that impair their health and quality of life. Despite the importance of nutrition in managing CP and the recognition of physiological, behavioral, and social causes of malnutrition, research on the effectiveness of individualized nutritional interventions developed and supported by multidisciplinary teams is scarce.

Aim

The study will evaluate the impact of an individualized nutritional intervention developed and supported by a multidisciplinary team on the anthropometric outcomes and overall health of children with CP.

Methods

A single-center, randomized controlled trial, conducted at the Medical University of Varna, Bulgaria, will enroll 100 children aged 2–12 years and diagnosed with CP. Participants will be randomly assigned to either an intervention group, receiving comprehensive structured dietary assessment and individualized nutrition plan developed by a multidisciplinary team of experts, or to a standard care group. Outcomes assessed will focus on anthropometric measures of nutritional status, but also include health outcomes, child development and clinical assessments, and quality of life indicators.

Ethics

Ethical approval for this study has been obtained from the Medical Ethics Committee at the Medical University of Varna (Protocol No. 134 dated 20.07.2023).

Conclusion

This study will assess the benefits of a multidisciplinary, individualized nutritional intervention for children with CP. The findings will have implications for clinical guidelines and interventions aiming to improve their care and quality of life.

Background

Time-to-event (TTE) endpoints are evaluated as the primary endpoint in single-arm clinical trials; however, limited options are available in statistical software for sample size calculation. In single-arm trials with TTE endpoints, the non-parametric log-rank test is commonly used. Parametric options for single-arm design assume survival times follow exponential distribution or Weibull distribution.

Methods

The exponential- or Weibull-distributed survival time assumption does not always reflect hazard pattern of real-life diseases. We therefore propose gamma distribution as an alternative parametric option for designing single-arm studies with TTE endpoints. We outline a sample size calculation approach using gamma distribution with a known shape parameter and explain how to extract the gamma shape estimate from previously published resources. In addition, we conduct simulations to assess the accuracy of the extracted gamma shape parameter and to explore the impact on sample size calculation when survival time distribution is misspecified.

Results

Our simulations show that if a previously published study (sample sizes $\ge$ 60 and censoring proportions $\le$ 20 %) reported median and inter-quartile range of survival time, we can obtain a reasonably accurate gamma shape estimate, and use it to design new studies. When true survival time is Weibull-distributed, sample size calculation could be underestimated or overestimated depending on the hazard shape.

Conclusions

We show how to use gamma distribution in designing a single-arm trial, thereby offering more options beyond the exponential and Weibull. We provide a simulation-based assessment to ensure an accurate estimation of the gamma shape and recommend caution to avoid misspecification of the underlying distribution.

Background

Child sexual abuse (CSA) affects 1 in 5 girls and 1 in 12 boys before age 18. Universal school-based prevention programs are an effective and cost-efficient method of teaching students an array of personal safety skills. However, the programmatic reach of universal school-based programs is limited by the inherent reliance on the school infrastructure and a dearth of available alternative delivery modalities.

Methods

The design for this study will use a rigorous cluster randomized design (N = 180 classrooms) to determine the equivalence of two delivery modalities of Safe Touches: as usual vs. modified. The as usual workshop will be delivered by two facilitators with live puppet skits (n = 90). Whereas, the modified workshop will be delivered by one facilitator using prerecorded skit videos (n = 90). We will determine the equivalence by measuring concept learning acquisition preworkshop to immediate postworkshop (Aim 1) and retention at 3-months postworkshop (Aim 2) among students in classrooms that receive the as usual or modified workshops. To conclude equivalence, it is imperative to also examine factors that may impact future dissemination and implementation, specifically program adoption among school personnel and implementation fidelity between the two modalities (Aim 3).

Conclusion

Study findings will inform the ongoing development of effective CSA prevention programs and policy decisions regarding the sustainable integration of such programs within schools.

Clinical trial registration

NCT06195852.

Introduction

Childhood specific phobias are among the most common and earliest onset mental disorders with a lifetime prevalence of more than ten percent. Brief intensive cognitive behavioral therapy (CBT) programs such as the One-Session Treatment (OST) are found to be effective in the remission of the specific phobias following treatment, but there is still room for improvement. The goal of the current study is to examine whether the long-term efficacy of OST increases by using a homework program supported by an app specifically designed for children; the Kids Beat Anxiety (KibA) homework program.

Methods

Children aged between 7 and 14 years with a specific phobia receive OST preceded by a three-week baseline phase to control for time-effects. Directly following OST, children are randomized to either a four-week homework period supported by an app (OST + app), or standard One-Session Treatment with a four-week homework period that is only supported by therapist instructions (OST-only). Primary outcome variables are diagnosis and severity of the specific phobia. Secondary outcomes include behavioral avoidance, self-reported fear, and functional impairment. Data will be analyzed based on intention-to-treat and per protocol samples using mixed-effects multilevel linear models.

Ethics and dissemination

The current study was approved by the METC of the Academic Medical Center, Amsterdam, The Netherlands (number: NL72697.018.20) and the Ethical Committee of the Ruhr University, Bochum, Germany (number: 663). Results of this trial will be published in peer-reviewed journals.

Trial registration

The study was pre-registered at the Dutch Trial Register, number: NL 9216.

Background

This pragmatic clinical trial aims to determine the efficacy and safety of add-on Astragalus membranaceus (AM) for cognition and non-cognition in patients with of mild to moderate Alzheimer's disease complicated with orthostatic hypotension in orthostatic hypotension, elucidate the underlying mechanisms, identify related response predictors, and explore effective drug components.

Methods

This is an add-on, assessor-blinded, parallel, pragmatic, randomized controlled trial. At least 66 adults with mild to moderate Alzheimer's disease (AD) and OH aged 50–85 years will be recruited. Participants will be randomized in a 1:1:1 ratio to receive 24 weeks of routine care or add-on low dose AM or add-on high dose AM group. The primary efficacy outcome will be measured by the Alzheimer's Disease Assessment Scale-Cognitive Subscale, Chinese version. Secondary efficacy outcome assessment will include neuropsychological tests, blood pressure, plasma biomarkers, multimodal electroencephalograms, and neuroimaging. Safety outcome measures will include physical examinations, vital signs, electrocardiography, laboratory tests (such as hematologic and blood chemical tests), and adverse event records.

Ethics and dissemination

This trial was approved and supervised by Fujian Medical University Union Hospital (2021KJCX040). Independent results, findings will be published in peer-reviewed journals and presented at national and international conferences.

Trial registration number

NCT05647473; ClinicalTrials.gov Identifier.

Introduction

The essential oil of Shikuwasa (Citrus depressa Hayata) primarily contains limonene and γ-terpinene, which have potential applications in stress management and relaxation. However, the psychological or physiological relaxation effects of Shikuwasa essential oil on humans are still unknown. This study aims to investigate the short-term relaxation effects of Shikuwasa essential oil, one of the less-studied varieties, compared to inhaling odour-free air in young female adults.

Methods

and analysis: This study is a two-arm, parallel-group, open-label, randomised controlled superiority trial. Forty young female adults will be assigned with a 1:1 allocation ratio to either the Shikuwasa essential oil inhalation group or the odour-free air inhalation group. The primary outcome measure will be subjective tense arousal (subscale of the Japanese version of the University of Wales Institute of Science and Technology Mood Adjective Checklist). Secondary outcomes include objective measures: miosis rate and peripheral skin temperature for evaluating autonomic nervous activity, and cerebral blood flow (assessed using near-infrared spectroscopy) for evaluating central nervous activity. Since these objective outcome measures cannot be performed at the same time, we divide our experiment into three phases and participants will inhale sample vials for 2 min in each experiment. We will also evaluate individual preferences/impressions regarding inhaled samples and any adverse events.

Ethics and dissemination

The study protocol has been reviewed and approved by the Research Ethics Committee of the Faculty of Medicine, University of Miyazaki (reference no: I-0074). The findings of this study will be disseminated to academic and professional audiences via publications in peer-reviewed journals and presentations at academic conferences, and to the broader public via public talks and media/press releases. All study findings, whether negative or positive, will be reported.

Trial registration

UMIN Clinical Trials Registry (UMIN-CTR), UMIN000053914. Prospectively registered on March 20, 2024.

Background

Poor long-term recovery outcomes after treatment (e.g., readmission to inpatient treatment) are common among individuals with substance use disorders (SUDs). In-person mindfulness-based treatments (MBTs) are efficacious for SUDs and may improve recovery outcomes. However, existing MBTs for SUD have limited public health reach, and thus scalable delivery methods are needed. A digitally-delivered MBT for SUDs may hold promise.

Methods

We recently developed Mindful Journey, a smartphone app-based adjunctive MBT for improving long-term recovery outcomes. In this paper, we present details on the app and describe the protocol for a single-site pilot feasibility randomized controlled trial of Mindful Journey. In this trial, individuals (n = 34) in an early phase of outpatient treatment for SUDs will be randomized to either treatment-as-usual (TAU) plus Mindful Journey, or TAU only. The trial will focus on testing the feasibility (e.g., engagement) and acceptability of the app (e.g., perceived usability and helpfulness for recovery), as well as feasibility of study procedures (e.g., assessment completion). The trial will incorporate ecological momentary assessment before and after treatment to assess mechanisms in real-time, including mindfulness, craving, difficulties with negative emotion regulation, and savoring. To examine the sensitivity to change of outcomes (substance use, substance-related problems, and psychological distress) and mechanism variables (noted above), we will test within-treatment-condition changes over time.

Discussion

The proposed pilot trial will provide important preliminary data on whether Mindful Journey is feasible and acceptable among individuals with SUDs.

Trial registration

ClinicalTrials.gov NCT05109507.

Stroke is a highly disabling condition, for which music therapy is regularly used in rehabilitation. One possible mechanism for the effects of music therapy is the motivational aspect of music, for example regarding treatment adherence based on improved mood. In this study, changes in motivation related to Neurologic Music Therapy (NMT) techniques during rehabilitation in the subacute phase after stroke will be investigated. Using a randomised within-subjects cross-over design, the effects of two NMT techniques and related motivational indices will be investigated. Data will be collected at three timepoints: baseline (TP1), after being randomised into groups and receiving NMT or standard care (TP2), and then at a third time point after the cross-over and having received both conditions (TP3). This design allows to counteract order effects, time effects due to spontaneous and/or nonlinear recovery, as well as single-subject comparisons in a relatively heterogeneous sample. Twenty adult participants who have experienced a supratentorial ischaemic or haemorrhagic stroke and are experiencing upper-limb impairments and/or cognitive deficits will be included. Behavioural measures of motor function, cognition, and quality of life will be collected, along with self-reported indices of overall motivation. The study outcomes will have implications for the understanding of the underlying mechanisms of music therapy in stroke recovery, more specifically regarding the relevance of motivational states in neurorehabilitation.

Background

Cognitive symptoms, among the core symptoms of schizophrenia, are associated with poor functional outcome and burden of illness. To date, there is no effective pharmacological treatment for these symptom clusters. Augmentation with psychostimulants has been proposed as a potential treatment option.

Objectives

The present study aims to assess off-label use of adjunctive methylphenidate extended release (ER) in patients with schizophrenia who are stable on antipsychotic medications, and to assess its efficacy on functioning and cognitive outcome.

Methods

This is a single centre study at the Royal Ottawa Mental Health Centre. An open-label fixed dose controlled cross-over trial is planned. Eligible participants will be randomized into one of two arms of the study: 1) four weeks of add-on methylphenidate ER 36 mg, or 2) four weeks of treatment as usual. At 4 weeks, participants will switch arms. The duration of the study includes 8 weeks of treatment and a follow-up visit at 12 weeks. Primary outcome measures include tablet-based tests of functioning and cognition (VRFCAT and BAC) and will be administered at baseline and every 4 weeks. We are aiming to recruit a total of 24 participants.

Expected outcomes

The proposed project intends to assess a potential treatment option for cognitive deficits of schizophrenia, for which there are no recommendations by current treatment guidelines. The novelty and significance of the current study is that it investigates this intervention and assess applicability of it in a “real world setting” in a tertiary care hospital.

Objectives

Methods

Results

Conclusion