Status:
RECRUITING
Safety and Dystrophin Expression of SPOT-mRNA03 in Duchenne Muscular Dystrophy (DMD) Patients
Lead Sponsor:
Shanghai Siponuoyin Biotechnology Co Ltd
Collaborating Sponsors:
Shanghai Children's Medical Center
Conditions:
Duchenne Muscular Dystrophy (DMD)
Eligibility:
MALE
2-6 years
Phase:
EARLY_PHASE1
Brief Summary
The primary objective of this study is to evaluate the safety and and tolerability of SPOT-mRNA03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily ...
Detailed Description
This is a FIH, open-label, single-arm, and single-center exploratory clinical study of SPOT-mRNA03 administered via IV infusion for DMD patients. SPOT-mRNA03 is a muscle-targeted extracellular vesicle...
Eligibility Criteria
Inclusion
- According to the requirements of the region/country and/or IRB/IEC, the patient and/or legal guardian have signed a written informed consent form and are aware of all relevant study content.
- Ambulatory boys aged between 2 to 6 years of age, inclusive who can work without assistance for at least 10 meters.
- The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome sequencing).
- Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy.
- Heart, liver, lung, and kidney functions are sufficient:
- The left ventricular ejection fraction (LVEF) should be ≥ 50%;
- Forced vital capacity (FVC) \> 50% of the expected value, and do not require nighttime ventilation;
- Patient's glomerular filtration rate (GFR)\>30 mL/min/1.73 m2
Exclusion
- Complications other than DMD that may cause muscle weakness and/or motor dysfunction.
- There are severe intellectual disabilities (such as severe autism, severe cognitive impairment, and severe behavioral disorders) that, according to the investigator's judgment, can affect the study.
- Hospitalization for respiratory failure within 8 weeks prior to screening.
- Asthma or underlying lung diseases that are poorly controlled, such as bronchitis, bronchiectasis, emphysema, or recurrent infectious pneumonia that investigator believes may affect respiratory function.
- Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions：
- Intravenous administration of diuretics or positive inotropic drugs is required within 8 weeks prior to screening.
- Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening.
- Abnormal laboratory values considered clinically significant:
- GGT \> 3 × upper limit of normal
- Bilirubin ≥ 3.0 mg/dL
- Creatinine ≥ 1.8 mg/dL
- Hemoglobin \< 8 or \> 18 g/dL
- White blood cell count \> 18,500/μL
- Arrhythmias that require anti-arrhythmic treatment.
- Subjects who are undergoing immunosuppressive therapy.
- Has used other gene therapy, investigational drugs, or any treatment aimed at increasing dystrophin expression.
- Subjects with a history of major surgeries within 12 weeks prior to the initial infusion or planning to undergo major surgeries (such as scoliosis surgery) during this study.
- Subjects who are allergic to investigational products or local aesthetic drugs or have a history of severe allergies or genetic allergic reactions.
- Within 6 months prior to the initial infusion, the subjects are exposed to another investigational drug or have participated in an intervention clinical trial.
- Subjects with positive hepatitis B core antibody or hepatitis C antibody or HIV antibody during screening.
- Investigator believes that the presence of any other serious diseases, medical conditions, or chronic drug treatment needs can pose unnecessary risks to gene transfer.
Key Trial Info
Start Date :
August 6 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT07188012
Start Date
August 6 2025
End Date
December 1 2026
Last Update
September 23 2025
Active Locations (1)
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1
Shanghai Children's Medical Center
Shanghai, Shanghai Municipality, China