The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-A101 to treat participants with knee osteoarthritis.
This study is divided into two phases: Phase Ia and Phase Ib . Phase Ia is a randomized, single-blind study . Phase Ib is a randomized, double blind, parallel assignment study. BBM-A101 is an AAVbased gene therapy that delivers a therapeutic gene cassette into knee for the treatment of knee osteoarthritis.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
81
Shenzhen Second People's Hospital
Shenzhen, Guangdong, China
RECRUITINGThe Ninth People's Hospital of Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
RECRUITINGIncidence of dose limiting toxicity (DLT) events
Time frame: within 4 weeks
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
To evaluate the safety of BBM-A101 by AEs and SAEs.
Time frame: within 52 weeks
The protein levels of gene expression products in blood and joint fluid
Time frame: within5 years
The shedding of recombinant adeno-associated virus (rAAV) vectors in peripheral blood, joint fluid, urine, feces, saliva, and semen
Time frame: within 5 years
The change in the joint space width as detected by X-ray of the knee joint compared to the baseline
Time frame: within 5 years
The changes in the titers of AAV capsid-binding antibodies in the blood and joint fluid compared to the baseline level
Time frame: within 5 years
The changes in the titers of antibodies binding to the expressed products in the blood and joint fluid compared to the baseline level
Time frame: within 5 years
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Placebo(phase Ib)