Condition: Hemophilia B · Sponsor: Institute of Hematology & Blood Diseases Hospital, China
This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
This description comes directly from the study's public registry record.
Feng Xue, MD · +862223909240 · xuefeng@ihcams.ac.cn
Shuo Chen, BS · +862223909009 · Chenshuo@ihcams.ac.cn
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| Institute of haematology and Blood diseases hospital | Tianjin, Tianjin Municipality, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05709288