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

Brief Title

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

Official Title

A Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B

Brief Summary

      This is a multi-center, 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.

Study Phase

Phase 1/Phase 2

Study Type

Interventional

Primary Outcome

Incidence of dose limiting toxicity (DLT) events.

Secondary Outcome

 FIX activity

Condition

Hemophilia B

Intervention

Single dose intravenous injection of BBM-H901

Study Arms / Comparison Groups

 Arm of BBM-H901

Description:  1×10^13 vg/kg, Single-dose treatment

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Genetic

Estimated Enrollment

Start Date

December 30, 2021

Completion Date

June 30, 2028

Primary Completion Date

June 30, 2024

Eligibility Criteria

Inclusion Criteria:

1. Males ≥ 18 years of age;

2. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels;

3. Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX
protein products based on historical data from the subjects' records/histories;

4. Have had bleeding events and/or injected with FIX protein products (including
recombination and plasma source) during the last 12 weeks documented in the subjects'
medical records;

5. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV
immunoglobulin administration;

6. Agree to use a reliable barrier contraception method from the beginning of signing the
informed consent to 52 weeks after administration.

Exclusion Criteria:

1. Being positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus-DNA
(HBV-DNA). Being positive for hepatitis C virus antibody (HCV-Ab) or hepatitis C virus
RNA (HCV-RNA). Subjects with medical history of hepatitis B or C can be regarded as
negative only when 2 required samplings are conducted at least 3 months apart and both
test results of indicators aforementioned are negative, i.e. subjects with natural
clearance and anti-viral therapy clearance for hepatitis B or C are eligible;

2. Have potential liver diseases, such as previous diagnosis of portal hypertension,
splenomegaly, hepatic encephalopathy or liver fibrosis (fibrosis stage ≥ 3); nodules
or cysts were found by B ultrasound, or elevated alpha-fetoprotein was detected by
laboratory tests. Subjects who are not eligible for the study if the abnormalities are
clinically significant regarding to the medical judgement of the investigator;

3. HIV positive patients;

4. Have participated in a previous gene therapy research trial before screening, or in a
clinical study with an investigational drug within 5 half-life of the investigational
product, whichever is longer;

5. Have alcohol or drug dependence, or cannot stop drinking throughout the study;

6. Any concurrent clinically significant major disease or condition that the investigator
deems unsuitable for participation in the study.

Gender

Male

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Lei Zhang, MD, 16602669908, [email protected]

Location Countries

China

Location Countries

China

Administrative Informations

NCT ID

NCT05203679

Organization ID

BBM001-CLN1001

Responsible Party

Sponsor

Study Sponsor

Shanghai Belief-Delivery BioMed Co., Ltd

Study Sponsor

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

Verification Date

January 2023