Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis

• Published in: Muscle & nerve Vol. 70; no. 2; pp. 232 - 239
• Main Authors: 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., Paganoni, Sabrina
• Format: Journal Article
• Language: English
• Published: Hoboken, USA John Wiley & Sons, Inc 01.08.2024; Wiley Subscription Services, Inc
• Subjects: Adult; Aged; Amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - drug therapy; Clinical trials; Design; Drug development; Drugs, Investigational - therapeutic use; expanded access; Female; Health Services Accessibility; Humans; investigational product; Male; Middle Aged; motor neuron disease; United States; United States Food and Drug Administration; United States; expanded access; motor neuron disease; amyotrophic lateral sclerosis; investigational product
• ISSN: 0148-639X; 1097-4598; 1097-4598
• DOI: 10.1002/mus.28169

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.