Duchenne Muscular Dystrophin (DMD)Early Phase 1MUSCLENCT06594094
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
A Early Phase 1 study testing HG302 in people with Duchenne Muscular Dystrophin (DMD), run by HuidaGene Therapeutics Co., Ltd. at 1 site in China.
Trial summary
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys. Read more about this study
- Who is it for?
- Ages 4 to 8 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophin (DMD)
- What is being tested?
- HG302
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteChina
- Study dates
- 6 Nov 2024to 2 Jun 2027
- 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 8 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Males ≥ 4 and ≤8 years at the time of signing informed consent, with clinical diagnosis of DMD;
- DMD gene mutation types are deletions in exons 52, 52-61, or 52-63;
- Able to walk at least 10 meters independently;
- Willing to cooperate with muscle biopsy test;
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Who cannot join
- Presence of active infection;
- Presence of DMD-associated cardiomyopathy manifestations;
- Respiratory insufficiency requiring invasive or non-invasive ventilation;
- Serious infections such as pneumonia, pyelonephritis, or meningitis within 4 weeks prior to receiving trial drug infusion;
- Prior central nervous system surgery within 6 months before enrolment;
- Use of any investigational drug, or exon-skipping drug (whether investigational or not) 6 months prior to Screening;
- Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation);
- Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.
Trial locations and contacts
1 site in 1 country.
China1 site
Shanghai Children s Medical Center Affiliated to Shanghai Jiao Tong University School of Medical
Shanghai, Shanghai Municipality
About this study
Official title: An Investigator-initiated Clinical Study Evaluating the CRISPR-hfCas12Max Gene Editing Therapy in the Treatment of Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys. These mutations abolish dystrophin production in the muscle, leading to dystrophin deficiency at the myofiber membrane, continued fiber degeneration, the need for assisted ventilation, respiratory inflammation, loss of walking ability in their teens, followed by respiratory and cardiac decline, and eventually premature death before the age of 30.
Currently, there are only glucocorticoids for the standard supportive therapy of DMD, which can improve disease symptoms but do not change the outcome of the disease, Three antisense oligonucleotide (ASOs) medicines have been approved to treat DMD with exon 45-55 hotspot region mutations. However, they can only restore trace amounts of dystrophin protein, which is insufficient to bring real clinical benefits. Gene replacement therapy has been approved using adeno-associated virus (AAV) vectors to deliver the "mini-dystrophin" gene. Yet, mini-dystrophin gene-expression versions of truncated dystrophin functionality are sacrificed and limited.
HG302 uses a single AAV vector to deliver the CRISPR/hfCas12Max DNA editing system in the human DMD exon 51 splice donor site. Preclinical studies have shown that a single intravenous injection of HG302 significantly restores dystrophin protein expression in muscle fibers and rescues their muscle function in humanized DMD mice to wild-type levels, with long-lasting and durable efficacy.
- Conditions
- Duchenne Muscular Dystrophin (DMD)
- Being tested
- HG302
- Keywords
- DMD, dystrophin, Gene-editing, HG302, CRISPR
- 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.
HG302Genetic
Once intravenous injection; The duration of the study is about 110 weeks for each subject, including a 6 weeks screening period, enrollment visit, treatment visit, and 104 weeks follow-up period.
| Study group | Role | What they receive |
|---|---|---|
| HG302 | Experimental | The study will enroll up to 2 dose cohorts Receives: Genetic: HG302 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of systemic adverse events
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
Measured: 26 weeks
Secondary outcomes (4)
Change from baseline in percentage of dystrophin positive fiber
Test percentage of dystrophin positive fiber to detection of dystrophin expression
Measured: 26 weeks
Change from baseline in dystrophin fiber intensity
Test dystrophin fiber intensity to detection of dystrophin expression
Measured: 26 weeks
Change from baseline in dystrophin expression in skeletal muscle (western blotting)
Test dystrophin expression
Measured: 26weeks
Change from baseline in North Star Ambulatory Assessment scale and 6 minutes' walk test
North Star Ambulatory Assessment scale documents motor performance in children, with total score range from 0-34, the higher score means better motor performance; 6 minutes walk test measures walking endurance, allowing subjects to walk continuously for 6 minutes at the fastest possible pace.
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start6 Nov 2024
- Primary completion2 Aug 2026 (estimated)
- Study completion2 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophin (DMD)Studies of the same condition, open and under way.
- 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
- Sequential
- Model description
- Two dosing cohort: Low dose :2\~3 subjects High dose: 2\~3 subjects
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, HuidaGene Therapeutics Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06594094
- Sponsor's protocol ID
- HG30201
- Acronym
- MUSCLE
- Status last verified
- Jul 2026
- First submitted
- 10 Sep 2024
- Last updated
- 3 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: We did not plan to share any IPD with other researchers, any additional information required to reanalyze the data reported in this paper is available from the lead contact upon request.
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator-initiated Clinical Study Evaluating the CRISPR-hfCas12Max Gene Editing Therapy in the Treatment of Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT06594094
- Acronym / sponsor ID
- MUSCLE / HG30201
- Conditions
- Duchenne Muscular Dystrophin (DMD)
- Keywords
- DMD, dystrophin, Gene-editing, HG302, CRISPR
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 4 participants (actual)
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 6 Nov 2024 (actual)
- Primary completion
- 2 Aug 2026 (estimated)
- Study completion
- 2 Jun 2027 (estimated)
- First posted
- 19 Sep 2024 (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.
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys. These mutations abolish dystrophin production in the muscle, leading to dystrophin deficiency at the myofiber membrane, continued fiber degeneration, the need for assisted ventilation, respiratory inflammation, loss of walking ability in their teens, followed by respiratory and cardiac decline, and eventually premature death before the age of 30.
Currently, there are only glucocorticoids for the standard supportive therapy of DMD, which can improve disease symptoms but do not change the outcome of the disease, Three antisense oligonucleotide (ASOs) medicines have been approved to treat DMD with exon 45-55 hotspot region mutations. However, they can only restore trace amounts of dystrophin protein, which is insufficient to bring real clinical benefits. Gene replacement therapy has been approved using adeno-associated virus (AAV) vectors to deliver the "mini-dystrophin" gene. Yet, mini-dystrophin gene-expression versions of truncated dystrophin functionality are sacrificed and limited.
HG302 uses a single AAV vector to deliver the CRISPR/hfCas12Max DNA editing system in the human DMD exon 51 splice donor site. Preclinical studies have shown that a single intravenous injection of HG302 significantly restores dystrophin protein expression in muscle fibers and rescues their muscle function in humanized DMD mice to wild-type levels, with long-lasting and durable efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Two dosing cohort: Low dose :2~3 subjects High dose: 2~3 subjects
| Arm | Type | Receives |
|---|---|---|
| HG302 | Experimental | Genetic: HG302The study will enroll up to 2 dose cohorts |
Who may qualify
- Ages
- 4 to 8 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Males ≥ 4 and ≤8 years at the time of signing informed consent, with clinical diagnosis of DMD;
- DMD gene mutation types are deletions in exons 52, 52-61, or 52-63;
- Able to walk at least 10 meters independently;
- Willing to cooperate with muscle biopsy test;
- Acceptable hematology, clinical chemistry, and urine laboratory parameters.
Key exclusion criteria (8 listed)
- Presence of active infection;
- Presence of DMD-associated cardiomyopathy manifestations;
- Respiratory insufficiency requiring invasive or non-invasive ventilation;
- Serious infections such as pneumonia, pyelonephritis, or meningitis within 4 weeks prior to receiving trial drug infusion;
- Prior central nervous system surgery within 6 months before enrolment;
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 |
|---|---|---|
| HG302Once intravenous injection; The duration of the study is about 110 weeks for each subject, including a 6 weeks screening period, enrollment visit, treatment visit, and 104 weeks follow-up period. | Genetic | HG302 |
Primary outcome
Incidence and severity of systemic adverse events
Time frame: 26 weeks
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
4 secondary outcomes
Change from baseline in percentage of dystrophin positive fiber
Time frame: 26 weeks
Change from baseline in dystrophin fiber intensity
Time frame: 26 weeks
Change from baseline in dystrophin expression in skeletal muscle (western blotting)
Time frame: 26weeks
Change from baseline in North Star Ambulatory Assessment scale and 6 minutes' walk test
Time frame: 26 weeks
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 3 Aug 2026.
- Individual participant dataSharing planned: No. We did not plan to share any IPD with other researchers, any additional information required to reanalyze the data reported in this paper is available from the lead contact upon request.
Related trials for Duchenne Muscular Dystrophin (DMD)
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A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
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A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
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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.
Study Director study director
HuidaGene Therapeutics Co., Ltd.
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 HG302 in people with Duchenne Muscular Dystrophin (DMD), run by HuidaGene Therapeutics Co., Ltd. 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 8 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 3 Aug 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 systemic adverse events (time frame: 26 weeks). 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 3 Aug 2026.