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RECRUITINGPhase 1INTERVENTIONAL

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

A 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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify: 1. Voluntarily consents to participate in this study and provides written willing to sign a consent form prior to the start of any study specific procedures. 2. ≥ 18 years of age at the time of willing to sign a consent form. 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. Who Should NOT Join This Trial: 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 weakened immune system virus, hepatitis C virus antibody or hepatitis B virus. 5. Pregnant or currently breastfeeding. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

RAG-17

RAG-17 is a therapeutic small interfering RNA (siRNA).

DRUG

Placebo

Placebo will be administered via intrathecal injection

Locations (3)

Beijing Tiantan Hospital
Beijing, China
West China Hospital of Sichuan University
Chengdu, China
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, China