Ignite Creation Date:
2024-10-26 @ 3:42 PM
Last Modification Date:
2024-10-26 @ 3:42 PM
Study NCT ID:
NCT06641895
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-10-08
Brief Title:
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients With Duchenne Muscular Dystrophy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Single-Arm Open-Label Single-Dose Clinical Study to Evaluate the Safety Tolerability and Efficacy of BBM-D101 Injection in Patients With Duchenne Muscular Dystrophy
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of the study is to assess the safety tolerability and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy
Detailed Description:
This is a single-arm open-label study to evaluate the safety tolerability efficacy pharmacokinetic pharmacodynamic and immune response of BBM-D101 within 52 weeks after a single intravenous infusion in DMD boys as well as the long-term safety and efficacy of BBM-D101 for up to 5 years post infusion
BBM-D101 is gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle
BBM-D101 is gene addition therapy based on engineered AAV delivery therapeutic protein gene cassette into muscle for treating DMD Therapeutic protein could mediate the dystrophin-associated protein complex to prevent muscular dystrophy and to rescue the function of muscle
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None