Gene therapy for teenagers with Hemophilia B

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1 pilot study evaluates the safety, tolerability, and efficacy of a single intravenous infusion of BBM-H901 in male patients aged 12-18 years with Hemophilia B and low levels of factor IX. The study will enroll nine subjects who will receive a dose of 5x10^12 vg/Kg of BBM-H901, an adeno-associated viral vector designed to enhance the expression of the human factor IX transgene. Participants will undergo screening assessments and will be monitored for safety over a period of 52 weeks, with long-term follow-up extending up to ten years to assess the treatment's effects. The study is open-label and non-randomized, focusing on a single dose administration.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Subjects and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;
2. Be male and 12≤ age \<18 years of age, body wight ≥ 50kg;
3. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is \>2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤2% FIX coagulant activity (FIX:C) ;
4. Had had ≥75 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subject's record/history;
5. With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid;
6. Subjects with bleeding episode and/ or FIX agents infusion events within 12 weeks prior to screening;
7. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
8. Have no measurable FIX inhibitor as assessed by laboratory; or documented no prior history of FIX inhibitor (family history of inhibitors will not exclude the subject) and no clinical signs or symptoms of decreased response to FIX administration;
9. Have acceptable laboratory values:

   1. Hemoglobin ≥11 g/dL ;
   2. Platelets ≥100,000 cells/μL;
   3. AST, ALT ≤1.5x upper limit of normal at the testing laboratory;
   4. Bilirubin ≤1.5x ULN ;
   5. glomerular filtration rate eGFR ≥ 60ml/min.
10. For those subjects with sexual maturity, subject and statutory guardian must know that subjects must agree to use reliable barrier contraception until 52 weeks;
11. with good compliance to the schedule of visit and fill in the subject diary.

Exclusion Criteria:

1. Hepatitis B surface antigen antibody (HBSAg-Ab) or HBV-DNA positive; hepatitis C antibody or HCV-RNA positive;
2. Currently on antiviral therapy for hepatitis B or C;
3. With coagulation disorders other than hemophilia B;
4. Had immunosuppressive therapy other than steroid and other suggested IST agents within 30 days prior to screening;
5. Had vaccine 30 days prior to screening or have scheduled vaccination plan during the study (up to 52 weeks);
6. Have significant underlying liver disease, as defined by a preexisting diagnosis of portal hypertension, splenomegaly, encephalopathy, etc; other liver conditions unsuitable to gene therapy judged by investigator;
7. Have surgery plan within 52 weeks after gene therapy;
8. Have history of chronic infection or high rish of infection that the Investigator considers to constitute an unacceptable risk;
9. Had participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the last 12 weeks;
10. Had any herb that may affect the liver function within 4 weeks prior to screening;
11. Have history of fatal bleeding episode, eg intracranial hemorrhage, etc;
12. Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study;

Where it's running

• Institute of haematology and Blood diseases hospital
  Tianjin, Tianjin Municipality, China Recruiting

Study contacts

• Principal investigator: Lei Zhang, MD — Insitute of haematology and blood diseases hospital, chinese academy of medical sciences
• Study coordinator: Feng Xue, MD
• Email: xuefeng@ihcams.ac.cn
• Phone: +862223909240

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

  This study tests a single and multiple doses of SR604 to see if it is safe and effective for adults with Hemophilia A or B, with or without inhibitors, and for those with congenital Factor VII deficiency.

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• Single-dose CSL222 gene therapy for adolescent males (12–17) with severe or moderately severe hemophilia B.

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• ATHNdataset registry for people with bleeding and clotting disorders

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  Recruiting American Thrombosis and Hemostasis Network · Hickory, North Carolina · NCT06820515

• Is synovial proliferation in haemophilia joints active or inactive?

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• Observational study of blood clotting and bleeding disorders in Chinese patients

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  Recruiting Peking University People's Hospital · Beijing, Beijing Municipality and 4 other locations · NCT06727669

• Comparing ANB-002 to standard treatment for hemophilia B

  This study is testing a new treatment called ANB-002 to see if it works as well as the standard treatment for adults with hemophilia B who have low FIX activity and no FIX inhibitors.

  Recruiting at 11 sites Phase 3 Biocad · Chelyabinsk and 13 other locations · NCT06700096

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.