Official Title
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
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.

Detailed Description

Treating healthcare professionals (HCPs) of participants who meet the inclusion/exclusion

criteria may request access by emailing medicineaccess@clinigengroup.com. The current EAP

structure does not limit participants to receive access at specific sites. Treating HCPs can

submit requests on behalf of participants by emailing Clinigen at the email address provided

below. Information on the EAP eligibility criteria, including participants and center

criteria, will be provided by Clinigen as part of the enrollment request process.

Available
Intermediate-size Population
Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Drug: Tofersen
Administered as intrathecal bolus injection
Other Name: BIIB067

Eligibility Criteria

Key Inclusion Criteria: - 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).

Key Exclusion Criteria: - 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.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Eligibility Gender
All
Eligibility Age
Minimum: 18 Years
Countries
United States
Locations

Research Site
San Francisco, California, 94114

Available

Research Site
Chicago, Illinois, 60611

Available

Research Site
Kansas City, Kansas, 66160

Available

Research Site
Boston, Massachusetts, 02114

Available

Research Site
Worcester, Massachusetts, 01655

Available

Research Site
Rochester, Minnesota, 55905

Available

Research Site
Saint Louis, Missouri, 63110

Available

Research Site
Lebanon, New Hampshire, 03766

Available

Research Site
Amherst, New York, 14226

Available

Research Site
New York, New York, 10032

Available

Research Site
Syracuse, New York, 13210

Available

Research Site
Philadelphia, Pennsylvania, 19104

Available

Contacts

Clinigen Customer Services
MedicineAccess@clinigengroup.com

Global Biogen Clinical Trial Center
clinicaltrials@biogen.com

Medical Director
Study Director
Biogen

NCT Number
MeSH Terms
Motor Neuron Disease
Sclerosis