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Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

NCT05203679 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-04-01

No results posted yet for this study

Summary

This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels.

BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX.

Conditions

  • Hemophilia B

Interventions

GENETIC

Single dose intravenous injection of BBM-H901

Single dose intravenous infusion of BBM-H901, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene in liver.

Sponsors & Collaborators

  • Shanghai Xinzhi BioMed Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Lei Zhang, MD · Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

  • Caifeng Yang, Master · Shanghai Xinzhi BioMed Co., Ltd.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-30
Primary Completion
2024-04-16
Completion
2028-06-30

Countries

  • China

Study Locations

More Related Trials

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 NCT05203679 on ClinicalTrials.gov