Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.
Document Type
Article
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.
Medical Subject Headings
Amyotrophic Lateral Sclerosis; Humans; United States; Male; Female; Middle Aged; Aged; Drugs, Investigational; United States Food and Drug Administration; Adult; Health Services Accessibility
Publication Date
8-1-2024
Publication Title
Muscle & nerve
ISSN
1097-4598
Volume
70
Issue
2
First Page
232
Last Page
239
PubMed ID
38842106
Digital Object Identifier (DOI)
10.1002/mus.28169
Recommended Citation
Neel, Dylan V; Baselga-Garriga, Clara; Benson, Molly; Keegan, Mackenzie; Chase, Marianne; D'Agostino, Derek; Drake, Kristin; Hagar, Jennifer Linn; Hasenoehrl, Meredith Gibbons; Kulesa-Kelley, Jennifer; Leite, Alex; Mohapatra, Silpa; Portaro, Susanna Marie; Pothier, Lindsay M; Rosenthal, Jesse; Sherman, Alexander V; Yu, Hong; McCaffrey, Alexandra; Ho, Doreen; Luppino, Sarah; Bedlack, Richard; Heitzman, Daragh; Ajroud-Driss, Senda; Katz, Jonathan; Felice, Kevin; Whitaker, Charles; Ladha, Shafeeq; Alameda, Gustavo; Locatelli, Eduardo; Qureshi, Irfan A; Hotchkin, Michael T; Hayden, Michael R; Cudkowicz, Merit E; Babu, Suma; Berry, James D; and Paganoni, Sabrina, "Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis." (2024). Neurology. 1882.
https://scholar.barrowneuro.org/neurology/1882