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Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

NCT06111638 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 55

Last updated 2026-03-17

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-H803 injection in severe Hemophilia A subjects.

BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.

Conditions

Interventions

GENETIC

Single dose intravenous injection of BBM-H803

Single dose intravenous infusion of BBM-H803, an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene in liver. The dose of BBM-H803 will be calculated according to participant's weight.

Sponsors & Collaborators

  • Shanghai Mianyi Biopharmaceutical Co., Ltd.

    collaborator INDUSTRY

  • 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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-03
Primary Completion
2027-05-30
Completion
2031-05-30

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

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 NCT06111638 on ClinicalTrials.gov