A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation

Sponsor
Ractigen Therapeutics.
Study ID
NCT06556394
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • RAG-17 — DRUG
    RAG-17 is a therapeutic small interfering RNA (siRNA).
  • Placebo — DRUG
    Placebo will be administered via intrathecal injection

Study Details

This is a Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation

Key Dates

First listed
Aug 16, 2024
Start date
Dec 24, 2024
Status verified
Jan 2025
Primary completion
Feb 28, 2026
Completion
Apr 30, 2026

Study Design

Enrollment
32 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: RAG-17
    RAG-17 is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation
  • Placebo Comparator: Placebo
    Placebo is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation

Primary Outcome Measure

Adverse Events(AEs) [ Time Frame: Before treatment and within 57 days after treatment ]

Central Contacts

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