An Early Phase 1 interventional study of HG302 in Duchenne Muscular Dystrophin (DMD), sponsored by HuidaGene Therapeutics Co., Ltd.. Active, not recruiting at 1 site in China. Open to male participants aged 4 Years to 8 Years. Per ClinicalTrials.gov, last updated 2026-08-03.
Sponsored by HuidaGene Therapeutics Co., Ltd. · Early Phase 1, Interventional, and Treatment
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.
473 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
This study's enrollment of 4 is below the median of 26 across 326 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →HuidaGene Therapeutics Co., Ltd. is the lead sponsor of 6 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
The study will enroll up to 2 dose cohorts
Genetic: HG302
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.
Incidence and severity of systemic adverse events
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
Time frame: 26 weeks
Change from baseline in percentage of dystrophin positive fiber
Test percentage of dystrophin positive fiber to detection of dystrophin expression
Time frame: 26 weeks
Change from baseline in dystrophin fiber intensity
Test dystrophin fiber intensity to detection of dystrophin expression
Time frame: 26 weeks
Change from baseline in dystrophin expression in skeletal muscle (western blotting)
Test dystrophin expression
Time frame: 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.
Time frame: 26 weeks
Plan to share: 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.
No publications or documents are linked to this record.
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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HuidaGene Therapeutics Co., Ltd.