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.
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.
Drug: Tofersen
Administered as intrathecal bolus injection
Other Name: BIIB067
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
Research Site
Anchorage, Alaska, 99508
Research Site
Los Angeles, California, 90033
Research Site
Orange, California, 92868
Research Site
San Diego, California, 92103
Research Site
San Francisco, California, 94114
Research Site
Chicago, Illinois, 60611
Research Site
Kansas City, Kansas, 66160
Research Site
Boston, Massachusetts, 02114
Research Site
Worcester, Massachusetts, 01655
Research Site
Ann Arbor, Michigan, 48109
Research Site
Detroit, Michigan, 48202
Research Site
Rochester, Minnesota, 55905
Research Site
Saint Louis, Missouri, 63110
Research Site
Lebanon, New Hampshire, 03766
Research Site
Amherst, New York, 14226
Research Site
New York, New York, 10032
Research Site
Syracuse, New York, 13210
Research Site
Philadelphia, Pennsylvania, 19104
Research Site
Austin, Texas, 78759
Clinigen Customer Services
MedicineAccess@clinigengroup.com
Global Biogen Clinical Trial Center
clinicaltrials@biogen.com
Medical Director
Study Director
Biogen