Trial Profile

First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 06 Mar 2026

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At a glance

27 Feb 2026 Phase changed from phase 1 to phase 1/2 (Phase 1b: Dose Escalation Phase 2: Cohort Expansion).
27 Feb 2026 Planned End Date changed from 26 Apr 2029 to 5 Jun 2031.
27 Feb 2026 Planned primary completion date changed from 26 Apr 2029 to 5 Jun 2031.

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