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First-in-human multiple ascending dose study of intrathecal ALN-SOD in SOD1-mutant ALS
jRCT2061240039Phase 1Recruiting
- Sponsor
- Regeneron Pharmaceuticals
- Start
- Feb 2025
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
Phase 1, randomised, double-blind trial in amyotrophic lateral sclerosis (ALS) comparing ALN-SOD intrathecal with Placebo intrathecal (2 arms). Primary endpoints: Incidence of treatment-emergent adverse events and Severity of treatment-emergent adverse events. Started Feb 2025; status: recruiting.
02
Drugs
ALN-SOD
03
Conditions
amyotrophic lateral sclerosis (ALS)
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