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

NCT06556394 | Recruiting Phase 1 FDA Drug

• 1 other identifier: RGN17-001
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 3

Brief 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

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Adverse Events(AEs)

  Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)

  Before treatment and within 57 days after treatment

Secondary Outcomes (11)

• Plasma Pharmacokinetic (PK) Parameter: AUC0-last

  Before treatment and within 48 hours after treatment

• Plasma Pharmacokinetic (PK) Parameter: AUC0-inf

  Before treatment and within 48 hours after treatment

• Plasma Pharmacokinetic (PK) Parameter: Cmax

  Before treatment and within 48 hours after treatment

• Plasma Pharmacokinetic (PK) Parameter: Tmax

  Before treatment and within 48 hours after treatment

• Plasma Pharmacokinetic (PK) Parameter: λz

  Before treatment and within 48 hours after treatment

Study Arms (2)

RAG-17

EXPERIMENTAL

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

Drug: RAG-17

Placebo

PLACEBO COMPARATOR

Placebo is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation

Drug: Placebo

Interventions

RAG-17 DRUG

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

RAG-17

Placebo DRUG

Placebo will be administered via intrathecal injection

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ractigen Therapeutics.: lead

Study Sites (3)

Beijing Tiantan Hospital

Beijing, China

RECRUITING

West China Hospital of Sichuan University

Chengdu, China

RECRUITING

The Second Affiliated Hospital Zhejiang University School of Medicine

Hangzhou, China

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Long-Cheng Li

CONTACT

+86 18051622388; lilc@ractigen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2024
• First Posted: August 16, 2024
• Study Start: December 24, 2024
• Primary Completion: February 1, 2026
• Study Completion: April 1, 2026
• Last Updated: January 8, 2025

Record last verified: 2025-01

Locations

CN (3)