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

NCT07282652 | Active Not Recruiting Early Phase 1

• 1 other identifier: RAG180101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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)

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  To evaluate the safety profile of RAG-18 by recording the frequency, nature, and severity of all adverse events and serious adverse events observed during the study. The relationship of these events to the study drug will be assessed.

  From Baseline up to the end of the study (Day 169)

Secondary Outcomes (4)

• Maximum Observed Plasma Concentration (Cmax) of RAG-18

  Day 1 and Day 85 (assessed at pre-dose, and multiple timepoints up to 168 hours post-dose)

• Time to Maximum Observed Plasma Concentration (Tmax) of RAG-18

  Day 1 and Day 85 (PK samples collected at pre-dose and multiple timepoints up to 168 hours post-dose)

• Area Under the Plasma Concentration-Time Curve (AUC) of RAG-18

  Day 1 and Day 85 (PK samples collected at pre-dose and multiple timepoints up to 168 hours post-dose)

• Terminal Half-Life (t1/2) of RAG-18

  Day 1 and Day 85 (PK samples collected at pre-dose and multiple timepoints up to 168 hours post-dose)

Study Arms (1)

RAG-18

EXPERIMENTAL

The study will employ a dose-escalation design, enrolling participants into sequential cohorts for up to four planned dose levels. If the highest planned dose is well-tolerated, a decision to explore further dose escalation may be made based on the overall safety and risk-benefit evaluation.

Drug: RAG-18

Interventions

RAG-18 DRUG

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

RAG-18

Eligibility Criteria

• Age: 4 Years - 15 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• In accordance with the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use - Good Clinical Practice (ICH-GCP) guidelines and local/national and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements, the participant and/or their legal guardian has signed the written informed consent form.
• Aged 4-15 years old.
• Male patient with Duchenne Muscular Dystrophy (DMD), able to provide a written diagnosis from a specialist and a verifiable genetic test report.
• Able to undergo examinations required by the study protocol, such as muscle biopsy, Magnetic Resonance Imaging (MRI), and tests for motor and pulmonary function.
• Any disease-related concomitant medications must be in compliance with the study's requirements.

You may not qualify if:

• Prior treatment for Duchenne Muscular Dystrophy (DMD), regardless of whether the drug is marketed or not.
• Body Mass Index (BMI) \> 22 kg/m² or body weight ≥ 50 kg.
• Unable to complete the motor function tests required by the protocol, including: North Star Ambulatory Assessment (NSAA), Time to Stand (TTSTAND), 4-Stair Climb (4SCV), and the 6-Minute Walk Test (6MWT).
• Cardiac function at screening within the following ranges:
• Left Ventricular Ejection Fraction (LVEF) \< 55% as measured by Cardiac Magnetic Resonance (CMR).
• QT interval corrected using Fridericia's formula (QTcF) \> 450 ms at screening, or has additional risk factors for Torsades de Pointes.
• Hematology and electrolyte parameters at screening within the following ranges:
• Platelets \< 100,000/μL.
• Hemoglobin \< 12 g/dL.
• Absolute Neutrophil Count \< 1500/μL.
• Any serum calcium, potassium, sodium, magnesium, or phosphorus levels outside the clinically acceptable range for Duchenne Muscular Dystrophy (DMD) patients.
• International Normalized Ratio (INR), Prothrombin Time (PT), Partial Thromboplastin Time (PTT), Activated Partial Thromboplastin Time (aPTT), or Fibrinogen outside the normal range.
• History of medical conditions affecting liver function, with abnormal indicators within 28 days prior to the first dose.
• Presence of severe cardiac, renal, or respiratory dysfunction, or other severe complications.
• Allergy to the study drug or any of its components, or to Magnetic Resonance Imaging (MRI) contrast agents.

Sponsors & Collaborators

• Ractigen Therapeutics.: collaborator
• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, 100730, China

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: December 15, 2025
• Study Start: December 12, 2025
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: January 16, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)