Help us determine the optimal timing of an experimental treatment for SOD1 ALS, and whether it can delay clinical onset
Active - Not Recruiting
Study Name
Biogen ATLAS Trial
PI
Biogen (sponsor). Academic Lead: Michael Benatar, MD, PhD.
Study Type
Interventional (double-blind and open-label phases)
Eligible Population
Part A (Natural History Run-In)
- Age 18+
- Carrier of a protocol-defined SOD1 mutation
- Clinically pre-symptomatic for ALS
- Plasma neurofilament light (NfL) level below protocol-defined threshold at time of screening
Study Purpose/Goals
To evaluate whether pre-symptomatic initiation of tofersen can delay the emergence of clinically manifest ALS and/or slow decline in function after emergence of clinically manifest ALS.
Procedures/Participation
Up to 6.5 years of participation
- Screening: up to 42 days
- Part A (Natural History Run-In): up to 6.5 years
- Treatment: up to 2 years
- Open label extension
Procedures
- Medical history and medication review
- Drug dosing (as applicable)
- Blood, urine and CSF collection
- Neurological exam
- Electrocardiogram
- Physical exam
- Surveys
- Breathing test
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Join our family - together, we can change the course of this disease.
Join our family
Together, we can change the course of this disease.
ALS Treatment
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