Duchenne Muscular Dystrophy (DMD)Early Phase 1NCT07282652
A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
A Early Phase 1 study testing RAG-18 in people with Duchenne Muscular Dystrophy (DMD), run by Peking Union Medical College Hospital at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 4 to 15 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- RAG-18
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 12 Dec 2025to Nov 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 15 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- Receipt of a live (attenuated) vaccine within 28 days prior to the first dose of the study drug.
- Use of any other investigational drug, whether for DMD or not, from 28 days prior to the first dose of the study drug until the end of the study.
- Any reason that, in the investigator's opinion, would prevent the participant from fully participating in and completing the study, including inability to comply with study procedures or treatment, and other relevant medical or mental health conditions.
- Presence of a severe concomitant condition or disease that, in the investigator's opinion, would place the participant at undue risk or interfere with the study, including but not limited to: known moderate or severe persistent asthma, a history of asthma in the past 2 years, or currently uncontrolled asthma of any classification (Note: participants with currently controlled intermittent asthma or controlled mild persistent asthma are permitted to enroll); requirement for oxygen therapy to maintain adequate blood oxygen saturation; history of Chronic Obstructive Pulmonary Disease (COPD) within 6 months prior to signing the Informed Consent Form (ICF).
- Participant has an unstable systemic disease as judged by the investigator, including but not limited to severe hepatic, renal, respiratory, or metabolic diseases requiring medication.
- Any other unspecified reason that, in the investigator's opinion, makes the participant unsuitable for enrollment.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Investigator-Initiated Study to Evaluate the Safety/Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
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)
- Being tested
- RAG-18
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAG-18Drug
RAG-18 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: RAG-18 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From Baseline up to the end of the study (Day 169)
Secondary outcomes (4)
Maximum Observed Plasma Concentration (Cmax) of RAG-18
To evaluate the pharmacokinetic profile of RAG-18 after intravenous infusion.
Measured: 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
To determine the time to reach the maximum observed plasma concentration of RAG-18.
Measured: 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
To determine the total drug exposure over time by calculating the Area Under the Plasma Concentration-Time Curve (AUC).
Measured: 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
To determine the terminal elimination half-life of RAG-18 in plasma.
Measured: Day 1 and Day 85 (PK samples collected at pre-dose and multiple timepoints up to 168 hours post-dose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2025
- Study start12 Dec 2025
- Primary completionNov 2026 (estimated)
- Study completionNov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Ractigen Therapeutics.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07282652
- Sponsor's protocol ID
- RAG180101
- Status last verified
- Dec 2025
- First submitted
- 17 Nov 2025
- Last updated
- 16 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator-Initiated Study to Evaluate the Safety/Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT07282652
- Acronym / sponsor ID
- RAG180101
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Collaborators
- Ractigen Therapeutics.
- Study start
- 12 Dec 2025 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 15 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RAG-18 | Experimental | Drug: RAG-18The 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. |
Who may qualify
- Ages
- 4 to 15 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (21 listed)
- 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:
- Hematology and electrolyte parameters at screening within the following ranges:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RAG-18RAG-18 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands | Drug | RAG-18 |
Primary outcome
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Baseline up to the end of the study (Day 169)
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.
4 secondary outcomes
Maximum Observed Plasma Concentration (Cmax) of RAG-18
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Day 1 and Day 85 (PK samples collected at pre-dose and multiple timepoints up to 168 hours post-dose)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing RAG-18 in people with Duchenne Muscular Dystrophy (DMD), run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From Baseline up to the end of the study (Day 169)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jan 2026.