Clinical trials: ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
Familial Amyotrophic Lateral Sclerosis
Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations
This study will test the safety, tolerability and pharmacokinetics of single doses of ISIS 333611 administered into the spinal canal as 12 hour infusions.
- Trial name
- SOD-1 (NCT01041222)
- Testing
- ISIS 333611
- Where
- 4 sites in United States
- Volunteers
- 33 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
Long-Term Evaluation of BIIB067 (Tofersen)
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation.
- Trial name
- NCT03070119
- Testing
- Tofersen
- Where
- 30 sites in United States, Belgium and 7 more
- Volunteers
- 139 people
- Sponsor
- Biogen