NCT07188012 Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
| NCT ID | NCT07188012 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Shanghai Siponuoyin Biotechnology Co Ltd |
| Condition | Duchenne Muscular Dystrophy (DMD) |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2025-08-06 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 9 participants in total. It began in 2025-08-06 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: 1. 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. 2. Boys aged ≥ 2 years to \< 8 years and capable of walking independently for at least 10 meters. 3. The medical history includes clinical diagnosis of DMD and confirmed Duchenne mutations using validated genetic testing (MLPA and whole genome sequencing). 4. Able to tolerate muscle biopsy under anesthesia and have no contraindications to biopsy. 5. Heart, liver, lung, and kidney functions are sufficient: 1. The left ventricular ejection fraction (LVEF) should be ≥ 50%; 2. Forced vital capacity (FVC) \> 50% of the expected value, and do not require nighttime ventilation; 3. Patient's glomerular filtration rate (GFR)\>30 mL/min/1.73 m2 Exclusion Criteria: 1. Complications other than DMD that may cause muscle weakness and/or motor dysfunction. 2. 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. 3. Hospitalization for respiratory failure within 8 weeks prior to screening. 4. 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. 5. Severe uncontrolled heart failure (NYHA III-IV), including any of the following conditions： 1. Intravenous administration of diuretics or positive inotropic drugs is required within 8 weeks prior to screening. 2. Hospitalization due to worsening heart failure or arrhythmia within 8 weeks prior to screening. 6. Abnormal laboratory values considered clinically significant: 1. GGT \> 3 × upper limit of normal 2. Bilirubin ≥ 3.0 mg/dL 3. Creatinine ≥ 1.8 mg/dL 4. Hemoglobin \< 8 or \> 18 g/dL 5. White blood cell count \> 18,500/μL 7. Arrhythmias that require anti-arrhythmic treatment. 8. Subjects who are undergoing immunosuppressive therapy. 9. Has used other gene therapy, investigational drugs, or any treatment aimed at increasing dystrophin expression. 10. 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. 11. Subjects who are allergic to investigational products or local aesthetic drugs or have a history of severe allergies or genetic allergic reactions. 12. Within 6 months prior to the initial infusion, the subjects are exposed to another investigational drug or have participated in an intervention clinical trial. 13. Subjects with positive hepatitis B core antibody or hepatitis C antibody or HIV antibody during screening. 14. Investigator believes that the presence of any other serious diseases, medical conditions, or chronic drug treatment needs can pose unnecessary risks to gene transfer.
Contact & Investigator
Wang Jiwen
PRINCIPAL INVESTIGATOR
Shanghai Children's Medical Center
Frequently Asked Questions
Who can join the NCT07188012 clinical trial?
This trial is open to male participants only, aged 2 Years or older, up to 7 Years, studying Duchenne Muscular Dystrophy (DMD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07188012 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07188012 currently recruiting?
Yes, NCT07188012 is actively recruiting participants. Contact the research team at Winston.town@spotbiosystems.com for enrollment information.
Where is the NCT07188012 trial being conducted?
This trial is being conducted at Shanghai, China.
Who is sponsoring the NCT07188012 clinical trial?
NCT07188012 is sponsored by Shanghai Siponuoyin Biotechnology Co Ltd. The principal investigator is Wang Jiwen at Shanghai Children's Medical Center. The trial plans to enroll 9 participants.