Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
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
Аннотация
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.