Safety and Dystrophin Expression of SPOT-mRNA03 in Duchenne Muscular Dystrophy (DMD) Patients

NCT07188012 | Recruiting Early Phase 1

• 1 other identifier: FM-T3-SH
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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 investigate the concentration changes in dystrophin mRNA concentration, dystrophin protein expression and engraftment, as well as cytokine profiles and immunogenicity.

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-mRNA03 in DMD patients

  Safety and tolerability of SPOT-mRNA03 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 6 months post-treatment

Secondary Outcomes (6)

• Concentration of Dystrophin mRNA in Serum

  16 weeks

• Concentration of Dystrophin mRNA in Muscles

  4 weeks

• Percent of Normal Dystrophin Protein Expression in Muscles

  4 weeks

• Percentage of Dystrophin-Positive Fibers in Muscles

  4 weeks

• Concentration of Cytokines in Serum

  28 weeks

Study Arms (1)

Low Dose and High Dose

OTHER

3 patients will be administered with low dose and 3 patients will be administered with high dose

Genetic: mRNA

Interventions

mRNA GENETIC

mRNA therapy delivered by extracellular vesicles (EVs)

Low Dose and High Dose

Eligibility Criteria

• Age: 2 Years - 6 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.
• 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

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

Interventions

RNA, Messenger

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

Intervention Hierarchy (Ancestors)

RNA; Nucleic Acids; Nucleic Acids, Nucleotides, and Nucleosides

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: 3 patients will receive a low dose and 3 patients will receive a high dose

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: September 23, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)