Actively Recruiting
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Led by Shanghai Siponuoyin Biotechnology Co Ltd · Updated on 2026-05-07
9
Participants Needed
1
Research Sites
68 weeks
Total Duration
On this page
Sponsors
S
Shanghai Siponuoyin Biotechnology Co Ltd
Lead Sponsor
S
Shanghai Children's Medical Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient or legal guardian has signed informed consent and understands the study
- Boys aged 2 years to less than 8 years who can walk independently for at least 10 meters
- Clinical diagnosis of Duchenne Muscular Dystrophy confirmed by genetic testing (MLPA and whole genome sequencing)
- Able to tolerate muscle biopsy under anesthesia with no contraindications
- Heart, liver, lung, and kidney functions sufficient:
- Left ventricular ejection fraction (LVEF) of 50% or higher
- Forced vital capacity (FVC) greater than 50% of expected value without need for nighttime ventilation
- Glomerular filtration rate (GFR) above 30 mL/min/1.73 m2
You will not qualify if you...
- Muscle weakness or motor dysfunction caused by conditions other than Duchenne Muscular Dystrophy
- Severe intellectual disabilities affecting participation, such as severe autism or cognitive impairment
- Hospitalization for respiratory failure within 8 weeks before screening
- Poorly controlled asthma or other underlying lung diseases affecting respiratory function
- Severe uncontrolled heart failure (NYHA III-IV), including recent intravenous diuretics or positive inotropic drugs use
- Hospitalization due to worsening heart failure or arrhythmia within 8 weeks before screening
- Clinically significant abnormal lab values including elevated GGT, bilirubin, creatinine, abnormal hemoglobin or white blood cell counts
- Arrhythmias requiring anti-arrhythmic treatment
- Current immunosuppressive therapy
- Prior use of gene therapy, investigational drugs, or treatments aimed at increasing dystrophin expression
- Major surgeries within 12 weeks before initial infusion or planned during the study
- Allergies to investigational products, local anesthetics, or history of severe allergic reactions
- Participation in another investigational drug trial or exposure within 6 months before infusion
- Positive for hepatitis B core antibody, hepatitis C antibody, or HIV antibody during screening
- Any other serious disease or condition posing unnecessary risk to gene transfer
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Center
Shanghai, Shanghai Municipality, China
Actively Recruiting
Research Team
W
Winston Town
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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