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New treatment option for Hemophilia B
Official title Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
ClinicalTrials.gov ID: NCT05709288
What this study is testing
What is BBM-H901?
BBM-H901 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia b.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 18, men only
You may be able to join if
- people and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;
- Be male and 12≤ age \<18 years of age, body wight ≥ 50kg;
- 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...
- 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...
- With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid;
You likely can't join if
- Hepatitis B surface antigen antibody (HBSAg-Ab) or HBV-DNA positive; hepatitis C antibody or HCV-RNA positive;
- Currently on antiviral therapy for hepatitis B or C;
- With coagulation disorders other than hemophilia B;
- Had immunosuppressive therapy other than steroid and other suggested IST agents within 30 days prior to screening;
- Had vaccine 30 days prior to screening or have scheduled vaccination plan during the study (up to 52 weeks);
- Have significant underlying liver disease, as defined by a preexisting diagnosis of portal hypertension, splenomegaly, encephalopathy, etc; other...
See the full eligibility criteria
- people and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;
- Be male and 12≤ age \<18 years of age, body wight ≥ 50kg;
- 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...
- 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;
- With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid;
- people with bleeding episode and/ or FIX agents infusion events within 12 weeks prior to screening;
- Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
- 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...
- Have acceptable laboratory values:
- Hemoglobin ≥11 g/dL ;
- Platelets ≥100,000 cells/μL;
- AST, ALT ≤1.5x upper limit of normal at the testing laboratory;
- Bilirubin ≤1.5x ULN ;
- glomerular filtration rate eGFR ≥ 60ml/min.
- For those people with sexual maturity, subject and statutory guardian must know that people must agree to use reliable barrier contraception until 52 weeks;
- with good compliance to the schedule of visit and fill in the subject diary.
- Hepatitis B surface antigen antibody (HBSAg-Ab) or HBV-DNA positive; hepatitis C antibody or HCV-RNA positive;
- Currently on antiviral therapy for hepatitis B or C;
- With coagulation disorders other than hemophilia B;
- Had immunosuppressive therapy other than steroid and other suggested IST agents within 30 days prior to screening;
- Had vaccine 30 days prior to screening or have scheduled vaccination plan during the study (up to 52 weeks);
- 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;
- Have surgery plan within 52 weeks after gene therapy;
- Have history of chronic infection or high rish of infection that the Investigator considers to constitute an unacceptable risk;
- 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;
- Had any herb that may affect the liver function within 4 weeks prior to screening;
- Have history of fatal bleeding episode, eg intracranial hemorrhage, etc;
- 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;
The study team makes the final eligibility decision.
Where it's taking place
- Tianjin, Tianjin Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 12 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Tianjin, Tianjin Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.