Amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1)
Conditions
Interventions
Research Name: BIIB067
Generic Name: Tofersen
Trade Names: Not applicable
Loading dose regimen for tofersen-naive participants only (doses 1 to 3): Tofersen will be administered on Days 0, 14, and 28.
Sponsors
Cho Kyo
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).
Exclusion criteria
Exclusion criteria: 1. Any comorbidities or conditions that, in the opinion of the Investigator, would unacceptably increase the risk of participation, including contraindications to lumbar punctures (LPs). 2. Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely held before and/or after an LP procedure according to local or institutional guidelines and/or Investigator determination. 3. Treatment with another investigational drug, biological agent (excluding tofersen), or device within 1 month or 5 half-lives of study agent, whichever is longer, before the first dose of tofersen in this study. 4. Female participants who are pregnant or breastfeeding. 5. Patient's primary place of residence is outside of the country of treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse event assessment | — |
Contacts
Public ContactBiogen Japan Medical Information
Biogen Japan Ltd.
Outcome results
None listed