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

NCT07188012 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-05-07

No results posted yet for this study

Summary

The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the changes in dystrophin nucleic acid concentration, dystrophin protein expression and engraftment, anti-dystrophin antibodies and cytokine profiles, as well as fat tissue mas and lean tissue mass following SPOT-03 administrations.

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Interventions

GENETIC

SPOT-03

SPOT-03 injection administered via IV infusion

Sponsors & Collaborators

  • Shanghai Children's Medical Center

    collaborator OTHER

  • Shanghai Siponuoyin Biotechnology Co Ltd

    lead INDUSTRY

Principal Investigators

  • Wang Jiwen · Shanghai Children's Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-06
Primary Completion
2026-12-31
Completion
2026-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07188012 on ClinicalTrials.gov