Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Global Early Access Program to Provide Tofersen To Patients With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene
1 other identifier
expanded_access
N/A
1 country
19
Brief Summary
The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
19 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 15, 2021
CompletedFirst Posted
Study publicly available on registry
July 22, 2021
CompletedJune 10, 2024
June 1, 2024
July 15, 2021
June 7, 2024
Conditions
Interventions
Eligibility Criteria
You may qualify if:
- Medically able to undergo the program procedures, as determined by the treating healthcare professional (HCP).
- Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).
You may not qualify if:
- Previous or current participation in a clinical trial of tofersen.
- Use of an investigational medicinal product (IMP) for amyotrophic lateral sclerosis (ALS) within 5 half-lives of the IMP before the first dose of tofersen.
- Participant's primary place of residence is outside of the country of treatment.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Biogenlead
Study Sites (19)
Research Site
Anchorage, Alaska, 99508, United States
Research Site
Los Angeles, California, 90033, United States
Research Site
Orange, California, 92868, United States
Research Site
San Diego, California, 92103, United States
Research Site
San Francisco, California, 94114, United States
Research Site
Chicago, Illinois, 60611, United States
Research Site
Kansas City, Kansas, 66160, United States
Research Site
Boston, Massachusetts, 02114, United States
Research Site
Worcester, Massachusetts, 01655, United States
Research Site
Ann Arbor, Michigan, 48109, United States
Research Site
Detroit, Michigan, 48202, United States
Research Site
Rochester, Minnesota, 55905, United States
Research Site
St Louis, Missouri, 63110, United States
Research Site
Lebanon, New Hampshire, 03766, United States
Research Site
Amherst, New York, 14226, United States
Research Site
New York, New York, 10032, United States
Research Site
Syracuse, New York, 13210, United States
Research Site
Philadelphia, Pennsylvania, 19104, United States
Research Site
Austin, Texas, 78759, United States
MeSH Terms
Interventions
Study Officials
- STUDY DIRECTOR
Medical Director
Biogen
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 15, 2021
First Posted
July 22, 2021
Last Updated
June 10, 2024
Record last verified: 2024-06