First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
Recruiting
Phase 1
Phase 2
Interventional Study
Amyotrophic Lateral Sclerosis (ALS)
Mutation in the Superoxide Dismutase-1 (SOD1) Gene
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 54 (estimated)
- Sponsor
- Regeneron Pharmaceuticals · Industry
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About This Trial
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people.
The aim of the study is to see how safe and tolerable the study…
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
1. Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol
2. Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in uprigh…
Contacts