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

Plain-language summary

This study evaluates the safety and efficacy of a gene therapy called BBM-H901, given as a single intravenous injection, in hemophilia B. It enrolled male patients aged 18 and older who have low levels of FIX clotting factor activity. It is being conducted in Phase 2/Phase 3 and is active but not currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE2/PHASE3

Status

Active, not recruiting

Sponsor

Shanghai Xinzhi BioMed Co., Ltd.

Intervention

Single dose intravenous injection of BBM-H901

Start date

12/30/2021

Estimated completion

6/30/2028

Countries

China

Related genes

Locations

• Hefei, China
• Beijing, China
• Guangzhou, China
• Shenzhen, China
• Tangshan, China
• Zhengzhou, China
• Taiyuan, China
• Tianjin, China
• Kunming, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria of Phase 1/2/3:

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 of Phase 1/2/3:

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.