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

amyotrophic lateral sclerosis expanded access investigational product motor neuron disease

Journal

Muscle & nerve
ISSN: 1097-4598
Titre abrégé: Muscle Nerve
Pays: United States
ID NLM: 7803146

Informations de publication

Date de publication:
06 Jun 2024
Historique:
revised: 03 05 2024
received: 29 11 2023
accepted: 12 05 2024
medline: 6 6 2024
pubmed: 6 6 2024
entrez: 6 6 2024
Statut: aheadofprint

Résumé

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. 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. 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. Multicenter EAPs conducted in parallel to randomized clinical trials for ALS can successfully enroll participants who do not qualify for clinical trials.

Identifiants

DOI: 10.1002/mus.28169 PMID: 38842106
pubmed: 38842106
doi: 10.1002/mus.28169
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : I AM ALS
Organisme : Biohaven Pharmaceuticals, Inc

Informations de copyright

© 2024 The Author(s). Muscle & Nerve published by Wiley Periodicals LLC.

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Auteurs

Dylan V Neel (DV)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
ORCID: 0000-0001-6964-9842

Clara Baselga-Garriga (C)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
ORCID: 0000-0001-7906-5752

Molly Benson (M)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Mackenzie Keegan (M)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Marianne Chase (M)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Derek D'Agostino (D)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Kristin Drake (K)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Jennifer Linn Hagar (JL)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Meredith Gibbons Hasenoehrl (MG)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Jennifer Kulesa-Kelley (J)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Alex Leite (A)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Silpa Mohapatra (S)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Susanna Marie Portaro (SM)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Lindsay M Pothier (LM)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Jesse Rosenthal (J)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Alexander V Sherman (AV)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Hong Yu (H)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Alexandra McCaffrey (A)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Doreen Ho (D)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Sarah Luppino (S)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Richard Bedlack (R)

Department of Neurology, Duke University, Durham, North Carolina, USA.
ORCID: 0000-0003-4666-7495

Daragh Heitzman (D)

MDA/ALS Clinic, Texas Neurology, Dallas, Texas, USA.

Senda Ajroud-Driss (S)

Les Turner ALS Center, Northwestern University, Chicago, Illinois, USA.

Jonathan Katz (J)

California Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California, USA.

Kevin Felice (K)

Department of Neuromuscular Medicine, Hospital for Special Care, New Britain, Connecticut, USA.

Charles Whitaker (C)

Department of Neuromuscular Medicine, Hospital for Special Care, New Britain, Connecticut, USA.

Shafeeq Ladha (S)

Gregory W. Fulton ALS and Neuromuscular Center, Barrow Neurological Institute, Phoenix, Arizona, USA.

Gustavo Alameda (G)

Holy Cross Hospital, Silver Spring, Maryland, USA.

Eduardo Locatelli (E)

Center for Collaborative Research, Nova Southeastern University and Dr. Kiran C. Patel College of Allopathic Medicine, Fort Lauderdale, Florida, USA.

Irfan A Qureshi (IA)

Biohaven Pharmaceuticals, Inc., New Haven, Connecticut, USA.

Michael T Hotchkin (MT)

Clene Nanomedicine, Inc., Salt Lake, Utah, USA.

Michael R Hayden (MR)

Naarden, Prilenia Therapeutics, The Netherlands.
Center for Molecular Medicine and Therapeutics, British Columbia Children's Hospital Research Institute, University of British Columbia, Vancouver, British Columbia, Canada.

Merit E Cudkowicz (ME)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
ORCID: 0000-0002-7075-1681

Suma Babu (S)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

James D Berry (JD)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Sabrina Paganoni (S)

Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Spaulding Rehabilitation Hospital, Harvard Medical School, Boston, Massachusetts, USA.
ORCID: 0000-0003-0505-1168

Classifications MeSH