NCT07282652

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

An Investigator-Initiated Study to Evaluate the Safety/Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: Male
• Age: 4 Years – 15 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy (DMD)

Interventions

Type	Name	Description
DRUG	RAG-18	RAG-18 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands

Timeline

• Start date: 2025-12-12
• Primary completion: 2026-11-01
• Completion: 2026-11-01
• First posted: 2025-12-15
• Last updated: 2026-01-16

Locations

1 site across 1 country: China