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Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

NCT06641895 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-03-25

No results posted yet for this study

Summary

The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Interventions

GENETIC

BBM-D101

BBM-D101 is a recombinant adeno-associated virus vector-based gene therapy for DMD treatment. It is a suspension for single intravenous (IV) infusion.

Sponsors & Collaborators

  • Shanghai Mianyi Biopharmaceutical Co., Ltd.

    collaborator UNKNOWN

  • Shanghai Jiao Tong University School of Medicine

    lead OTHER

Principal Investigators

  • Jiwen Wang · Shanghai Children's Medical Center, affiliated to Shanghai Jiao Tong University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
8 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-25
Primary Completion
2026-07-31
Completion
2030-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06641895 on ClinicalTrials.gov