The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
BBM-D101 is a recombinant adeno-associated virus vector-based gene therapy for DMD treatment. It is a suspension for single intravenous (IV) infusion.
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
RECRUITINGIncidence of dose limiting toxicity (DLT) events
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-D101 injection infusion.
Time frame: 12 weeks
The incidence of adverse events (AEs) and serious adverse events (SAEs)
To assess the safety of BBM-D101 Injection by AEs and SAEs.
Time frame: 52 weeks
Changes from baseline in the North Star Ambulatory Assessment (NSAA)
To assess changes in NSAA from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion; The NSAA is a scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. The NSAA total score is defined as the sum of all 17items, ranging from 0 (worst) to 34 (best).
Time frame: 52 weeks
Changes from baseline in the time to ascend 10-meter walk/run test (10MWR) without assistance
To assess changes in 10MWR from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Time frame: 52 weeks
Changes from baseline in the time to ascend time to rise (TTR) without assistance without assistance
To assess changes in TTR from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Time frame: 52 weeks
Changes from baseline in the time to ascend 4 steps (4-stair climb, 4SC) without assistance
To assess changes in 4-SC from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
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Time frame: 52 weeks
Changes from baseline in the time to ascend 100-meter walk/run test (100MWR) without assistance
To assess changes in 100MWR from baseline within 12 weeks, 26 weeks, and 52 weeks after BBM-D101 injection infusion
Time frame: 52 weeks
Changes from baseline in BBM-D101 genome copies in muscle biopsy samples
To assess changes of BBM-D101 genome from baseline in muscle in 12 weeks and 52 weeks following BBM-D101 administration. BBM-D101 genome was detected by Quantitative Polymerase Chain Reaction (QPCR).
Time frame: 52 weeks
Changes from baseline in BBM-D101 therapeutic protein level in muscle biopsy samples
To assess changes of BBM-D101 therapeutic protein from baseline in muscle in 12 weeks and 52 weeks following BBM-D101 administration. BBM-D101 therapeutic protein was detected by western blot (Jess) and tissue immunofluorescence.
Time frame: 52 weeks