NCT07188012

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

A Pilot Study for the Safety and Expression of Dystrophin in Skeletal Muscle After SPOT-mRNA03 Administration in Duchenne Muscular Dystrophy (DMD) Patients

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Shanghai Siponuoyin Biotechnology Co Ltd · Industry
• Sex: Male
• Age: 2 Years – 6 Years
• Healthy volunteers: Not accepted

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.

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 vesicles (EVs) loaded with full-length dystrophin mRNA. The targeting ability of SPOT-mRNA03 is conferred by molecular targeting peptides on the EVs membrane, enabling the delivery of dystrophin mRNA as a gene therapy product for DMD. The study has two ascending dose cohorts, 5.0 × 10\^9 CN dystrophin mRNA / kg and 5.0 × 10\^10 CN dystrophin mRNA / kg. The study will have a screening period of 30 days, during which patients or their legal guardian written informed consent will be obtained before screening assessments and eligibility will be determined. A total of 6 eligible subjects will participate in the study with 3 subjects in each dose cohort \[No previous treatment with corticosteroids\]. All subjects started taking 0.05-0.1mg/kg (adjusted according to the actual clinical situation) tacrolimus or sirolimus orally once daily at D-3 (3 days before initial dose of SPOT-mRNA03) for 4 weeks. All subjects are first administered via intravenous infusion on D1 and then administered twice a week (once every 4 days) for a total of 8 doses. Four weeks after the initial administration of the subjects in the previous dose cohort, if there are no serious adverse events related to the treatment, it will be determined that the subjects in next dose cohort could be administered after discussion between the investigators and the sponsor. Safety evaluations on subjects are conducted during each administration and follow-up.

Conditions

• Duchenne Muscular Dystrophy (DMD)

Interventions

Type	Name	Description
GENETIC	mRNA	mRNA therapy delivered by extracellular vesicles (EVs)

Timeline

• Start date: 2025-08-06
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2025-09-23
• Last updated: 2025-09-23

Locations

1 site across 1 country: China