Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.
| Title: | Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis. |
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| Authors: | 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; 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.; Bedlack R; Department of Neurology, Duke University, Durham, North Carolina, USA.; Heitzman D; MDA/ALS Clinic, Texas Neurology, Dallas, Texas, USA.; Ajroud-Driss S; Les Turner ALS Center, Northwestern University, Chicago, Illinois, USA.; Katz J; California Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California, USA.; Felice K; Department of Neuromuscular Medicine, Hospital for Special Care, New Britain, Connecticut, USA.; Whitaker C; Department of Neuromuscular Medicine, Hospital for Special Care, New Britain, Connecticut, USA.; Ladha S; Gregory W. Fulton ALS and Neuromuscular Center, Barrow Neurological Institute, Phoenix, Arizona, USA.; Alameda G; Holy Cross Hospital, Silver Spring, Maryland, USA.; Locatelli E; Center for Collaborative Research, Nova Southeastern University and Dr. Kiran C. Patel College of Allopathic Medicine, Fort Lauderdale, Florida, USA.; Qureshi IA; Biohaven Pharmaceuticals, Inc., New Haven, Connecticut, USA.; Hotchkin MT; Clene Nanomedicine, Inc., Salt Lake, Utah, USA.; 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.; 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.; 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.; 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.; 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. |
| Source: | Muscle & nerve [Muscle Nerve] 2024 Aug; Vol. 70 (2), pp. 232-239. Date of Electronic Publication: 2024 Jun 06. |
| Publication Type: | Journal Article; Multicenter Study |
| Language: | English |
| Journal Info: | Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE |
| Imprint Name(s): | Publication: : Hoboken, NJ : John Wiley & Sons; Original Publication: New York, NY : John Wiley & Sons |
| MeSH Terms: | Amyotrophic Lateral Sclerosis*/drug therapy; Drugs, Investigational/therapeutic use ; Humans ; United States ; Male ; Female ; Middle Aged ; Aged ; United States Food and Drug Administration ; Adult ; Health Services Accessibility ; Adaptive Clinical Trials as Topic |
| 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.; (© 2024 The Author(s). Muscle & Nerve published by Wiley Periodicals LLC.) |
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| Grant Information: | T32 GM144273 United States GM NIGMS NIH HHS; I AM ALS; Biohaven Pharmaceuticals, Inc |
| Contributed Indexing: | Keywords: amyotrophic lateral sclerosis; expanded access; investigational product; motor neuron disease |
| Substance Nomenclature: | 0 (Drugs, Investigational) |
| Entry Date(s): | Date Created: 20240606 Date Completed: 20240703 Latest Revision: 20250720 |
| Update Code: | 20260130 |
| DOI: | 10.1002/mus.28169 |
| PMID: | 38842106 |
| Database: | MEDLINE |
Journal Article; Multicenter Study