Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Conditions
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.
Interventions
DRUGTofersen
Administered as intrathecal bolus injection
Sponsors
Biogen
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Inclusion 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/
Countries
United States
Outcome results
None listed