Clinical Trial

A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy

Active, Not Recruiting Early Phase 1

Summary

This is an open-label, single-arm, dose-escalation trial to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular Dystrophy (DMD). The study will enroll approximately 12 subjects into four cohorts to assess the safety and tolerability of ascending intravenous doses. Secondary objectives include characterizing the pharmacokinetics (PK)/pharmacodynamics (PD) profile and assessing exploratory efficacy through changes in muscle biomarkers, muscle composition, cardiac/pulmonary function, and motor performance. The decision to escalate to the next dose level will be based on a comprehensive safety evaluation of the preceding cohort.

Protocol Amendment History 2 amendments

This ClinicalTrials.gov record has been amended 2 times since 2025-12-01; most recent amendment 2026-01-14.

Status change: Not Yet Recruiting → Active, Not Recruiting 2026-01-14

Trial Details

NCT Number	NCT07282652
Lead Sponsor	Peking Union Medical College Hospital Collaborators: Ractigen Therapeutics.
Conditions	Duchenne Muscular Dystrophy (DMD)
Enrollment	12 participants
Start Date	2025-12-12
Primary Completion	2026-11 (estimated)
Study Completion	2026-11 (estimated)
Updated on ClinicalTrials.gov	2026-01-16