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NCT07188012

Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients

StatusRecruiting
PhaseEarly Phase 1
Primary completion (estimated)

At risk: Primary completion moved at least 1 year 11 months later.

See at-risk trials from Shanghai Siponuoyin Biotechnology Co Ltd

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Record History

All 3 versions: 3 with changes below. See every version on ClinicalTrials.gov ↗

  1. Primary and study completion pushed from Dec 2026 to Dec 2028 Show 4 more changesShow less
    • Eligibility criteria expanded, about 26 words
    • Secondary endpoints: details revised at 6 of 7 entries
    • Site details revised
    • Collaborators: details revised at 1 of 1 entry
  2. Enrollment target: 6 to 9v2 Show 6 more changesShow less
    • Primary endpoint revised
    • Interventions: mRNA (Genetic) to SPOT-03 (Genetic)
    • Secondary endpoints: 6 to 7
    • Study description expanded, about 66 words
    • Study title revised
    • Eligibility criteria shortened, about 2 words
  3. Study First Submitted
  • Notable: the plan changed

Endpoints

Primary

Number of Participants with Treatment-Related Adverse Events Following Intravenous (IV) Infusion of SPOT-03 in DMD patients
Timeframe: From enrollment through 1-year post-treatment

Secondary (7)

Concentration of Dystrophin Nucleic Acid in Blood
Concentration of Dystrophin Nucleic Acid in Muscles
Percent of Normal Dystrophin Protein Expression in Muscles
Percentage of Dystrophin-Positive Fibers in Muscles
Concentration of Cytokines in Serum
Show 2 moreShow less
Concentration of Anti-Dystrophin Antibody in Serum
Adipose Tissue and Lean Tissue Mass (only Group II)

Arms and interventions

8-Dose and 32-DoseExperimental

In Group I, 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 8 doses. In Group II, at least 3 patients will be administered SPOT-03 by intravenous infusion on D1, followed by twice a week administration (once every 4 days) for a total of 32 doses.

Genetic: SPOT-03

Sites

1 site in 1 country

  • Shanghai Children's Medical CenterShanghai, Shanghai Municipality, China