Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT05454774
EARLY_PHASE1

A Study of FVIII Gene Therapy for Hemophilia A

Sponsor: Institute of Hematology & Blood Diseases Hospital, China

View on ClinicalTrials.gov

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.

Official title: A Clinical Study of AAV Vector Expressing Human Coagulation Factor FVIII Gene Therapy for Hemophilia A

Key Details

Gender

MALE

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

8

Start Date

2022-07-19

Completion Date

2027-09-15

Last Updated

2025-02-21

Healthy Volunteers

No

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

Locations (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China