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A Study of FVIII Gene Therapy for Hemophilia A

NCT05454774 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2025-02-21

No results posted yet for this study

Summary

This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) .

BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.

Conditions

Interventions

GENETIC

Single dose intravenous injection of BBM 002

Single dose intravenous infusion of BBM 002, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene in liver. The dose of BBM 002 will be 1×10\^13 vg/ kg

Sponsors & Collaborators

  • Institute of Hematology & Blood Diseases Hospital, China

    lead OTHER

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
2022-07-19
Primary Completion
2024-12-15
Completion
2027-09-15

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 NCT05454774 on ClinicalTrials.gov