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
RAG-17: RAG-17 is a therapeutic small interfering RNA (siRNA).
Placebo: Placebo will be administered via intrathecal injection
Study summary
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
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.
2. ≥ 18 years of age at the time of informed consent.
3. Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.
4. Documented SOD1 mutation.
5. Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).
6. If taking riluzole or edaravone, subject must be on a stable dose or ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
Exclusion Criteria:
1. Documented p.F21C SOD1 mutation.
2. Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.
3. Current enrollment in any other interventional study.
4. History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
5. Pregnant or currently breastfeeding.
Primary outcome measure(s)
Adverse Events(AEs) — Before treatment and within 57 days after treatment Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)
Trial sites (3)
Facility
City
Region
Status
Beijing Tiantan Hospital
Beijing
China
Recruiting
West China Hospital of Sichuan University
Chengdu
China
Recruiting
The Second Affiliated Hospital Zhejiang University School of Medicine
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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