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Recruiting NCT06556394

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

Phase I Interventional Amyotrophic Lateral Sclerosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: RAG-17, Placebo.
Who it may be relevant to
Registry conditions: Amyotrophic Lateral Sclerosis. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Detailed description

The study is a phase 1, randomized, double-blind, placebo controlled study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RAG-17 in patients with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) gene mutation. The dose levels will be evaluated sequentially across separate cohorts using a rules-based design, wherein participants will receive RAG-17 or placebo at a ratio of 3:1.

Interventions

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

Primary outcome measures

  • Adverse Events(AEs) [Time frame: Before treatment and within 57 days after treatment]
Secondary outcome measures (11)
  • Plasma Pharmacokinetic (PK) Parameter: AUC0-last [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: AUC0-inf [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: Cmax [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: Tmax [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: λz [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: T½ [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: CL/F [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: Vz/F [Time frame: Before treatment and within 48 hours after treatment]
  • Plasma Pharmacokinetic (PK) Parameter: MRT [Time frame: Before treatment and within 48 hours after treatment]
  • CSF Pharmacokinetic (PK) Parameter: Concentration [Time frame: Before treatment and within 29 days after treatment]
  • CSF Pharmacokinetic (PK) Parameter: T½ [Time frame: Before treatment and within 29 days after treatment]

Eligibility criteria

Inclusion criteria

  • Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.
  • ≥ 18 years of age at the time of informed consent.
  • Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.
  • Documented SOD1 mutation.
  • Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).
  • 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

  • Documented p.F21C SOD1 mutation.
  • 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.
  • Current enrollment in any other interventional study.
  • History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
  • Pregnant or currently breastfeeding.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Double blind
Primary purpose
Treatment

Study locations

China · 3 centers
  • Beijing Tiantan Hospital — Beijing
  • West China Hospital of Sichuan University — Chengdu
  • The Second Affiliated Hospital Zhejiang University School of Medicine — Hangzhou

Identifiers

NCT: NCT06556394 · RGN17-001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗