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

NCT07188012 | Recruiting Early Phase 1

• 1 other identifier: FM-T3-SH
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy; North Star Ambulatory Assessment; Dystrophin; Gene Therapy; Extracellular Vesicles

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Related Adverse Events Following Intravenous (IV) Infusion of SPOT-03 in DMD patients

  Safety and tolerability of SPOT-03 will be assessed by collection and quantification of all adverse events, graded according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

  From enrollment through 1-year post-treatment

Secondary Outcomes (7)

• Concentration of Dystrophin Nucleic Acid in Blood

  70 weeks

• Concentration of Dystrophin Nucleic Acid in Muscles

  16 weeks

• Percent of Normal Dystrophin Protein Expression in Muscles

  16 weeks

• Percentage of Dystrophin-Positive Fibers in Muscles

  16 weeks

• Concentration of Cytokines in Serum

  70 weeks

Study Arms (1)

8-Dose and 32-Dose

EXPERIMENTAL

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

Interventions

SPOT-03 GENETIC

SPOT-03 injection administered via IV infusion

8-Dose and 32-Dose

Eligibility Criteria

• Age: 2 Years - 7 Years
• Gender Eligibility Details: Male patients only, as Duchenne muscular dystrophy (DMD) predominantly affects males
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Boys aged ≥ 2 years to \< 8 years and capable of walking independently 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shanghai Siponuoyin Biotechnology Co Ltd: lead
• Shanghai Children's Medical Center: collaborator

Study Sites (1)

Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, China

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Wang Jiwen

  Shanghai Children's Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Winston Town

CONTACT

212-920-5501; Winston.town@spotbiosystems.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: August 22, 2025
• First Posted: September 23, 2025
• Study Start: August 6, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: May 7, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)