Dylan V Neel, Clara Baselga-Garriga, Molly Benson, Mackenzie Keegan, Marianne Chase, Derek D'Agostino, Kristin Drake, Jennifer Linn Hagar, Meredith Gibbons Hasenoehrl, Jennifer Kulesa-Kelley, Alex Leite, Silpa Mohapatra, Susanna Marie Portaro, Lindsay M Pothier, Jesse Rosenthal, Alexander V Sherman, Hong Yu, Alexandra McCaffrey, Doreen Ho, Sarah Luppino, Richard Bedlack, Daragh Heitzman, Senda Ajroud-Driss, Jonathan Katz, Kevin Felice, Charles Whitaker, Shafeeq Ladha, Gustavo Alameda, Eduardo Locatelli, Irfan A Qureshi, Michael T Hotchkin, Michael R Hayden, Merit E Cudkowicz, Suma Babu, James D Berry, Sabrina Paganoni
{"title":"Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.","authors":"Dylan V Neel, Clara Baselga-Garriga, Molly Benson, Mackenzie Keegan, Marianne Chase, Derek D'Agostino, Kristin Drake, Jennifer Linn Hagar, Meredith Gibbons Hasenoehrl, Jennifer Kulesa-Kelley, Alex Leite, Silpa Mohapatra, Susanna Marie Portaro, Lindsay M Pothier, Jesse Rosenthal, Alexander V Sherman, Hong Yu, Alexandra McCaffrey, Doreen Ho, Sarah Luppino, Richard Bedlack, Daragh Heitzman, Senda Ajroud-Driss, Jonathan Katz, Kevin Felice, Charles Whitaker, Shafeeq Ladha, Gustavo Alameda, Eduardo Locatelli, Irfan A Qureshi, Michael T Hotchkin, Michael R Hayden, Merit E Cudkowicz, Suma Babu, James D Berry, Sabrina Paganoni","doi":"10.1002/mus.28169","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction/aims: </strong>Expanded access (EA) is a Food and Drug Administration-regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers.</p><p><strong>Methods: </strong>A central coordination center was established to design and conduct the program. Templated documents and processes were developed to streamline study design, regulatory submissions, and clinical operations across protocols. The program included three protocols and provided access to IPs that were being tested in respective regimens of the HEALEY ALS Platform Trial (verdiperstat, CNM-Au8, and pridopidine). Clinical and safety data were collected in all EA protocols (EAPs). The program cohorts comprised participants who were not eligible for the platform trial, including participants at advanced stages of disease progression and with long disease duration.</p><p><strong>Results: </strong>A total of 85 participants were screened across the 3 EAPs from July 2021 to September 2022. The screen failure rate was 3.5%. Enrollment for the regimens of the platform trial was completed as planned and results informed the duration of the corresponding EAP. The verdiperstat EAP was concluded in December 2022. Mean duration of participation in the verdiperstat EAP was 5.8 ± 4.1 months. The CNM-Au8 and pridopidine EAPs are ongoing.</p><p><strong>Discussion: </strong>Multicenter EAPs conducted in parallel to randomized clinical trials for ALS can successfully enroll participants who do not qualify for clinical trials.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":null,"pages":null},"PeriodicalIF":2.8000,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Muscle & Nerve","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/mus.28169","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/6/6 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Introduction/aims: Expanded access (EA) is a Food and Drug Administration-regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers.
Methods: A central coordination center was established to design and conduct the program. Templated documents and processes were developed to streamline study design, regulatory submissions, and clinical operations across protocols. The program included three protocols and provided access to IPs that were being tested in respective regimens of the HEALEY ALS Platform Trial (verdiperstat, CNM-Au8, and pridopidine). Clinical and safety data were collected in all EA protocols (EAPs). The program cohorts comprised participants who were not eligible for the platform trial, including participants at advanced stages of disease progression and with long disease duration.
Results: A total of 85 participants were screened across the 3 EAPs from July 2021 to September 2022. The screen failure rate was 3.5%. Enrollment for the regimens of the platform trial was completed as planned and results informed the duration of the corresponding EAP. The verdiperstat EAP was concluded in December 2022. Mean duration of participation in the verdiperstat EAP was 5.8 ± 4.1 months. The CNM-Au8 and pridopidine EAPs are ongoing.
Discussion: Multicenter EAPs conducted in parallel to randomized clinical trials for ALS can successfully enroll participants who do not qualify for clinical trials.
期刊介绍:
Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.