Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Know the risks and potential benefits of clinical studies and talk to your health care provider before participating.
Read our disclaimer for details.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
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).
Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) decline faster than 2.0 points per month (points/month) at most recent assessment, with slope calculated as: [(48) - (ALSFRS-R score at entry)] / [months since symptom onset].
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