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New treatment option for Hemophilia A
Official title A Study of FVIII Gene Therapy for Hemophilia A
ClinicalTrials.gov ID: NCT05454774
What this study is testing
What is Single dose intravenous injection of BBM 002?
Single dose intravenous injection of BBM 002 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia a.
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 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older, men only
You may be able to join if
- people are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent.
- Males ≥ 18 years of age.
- Have hemophilia A with ≤2 IU/dL (≤2 %) endogenous FVIII activity levels.
- Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
- Have had bleeding events and/or infusions with FVIII protein products (including recombination and plasma source) during the last 12 weeks documented...
You likely can't join if
- 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...
- Currently on antiviral therapy for hepatitis B or C.
- Patients with coagulation disorders in addition to hemophilia A.
- Use of any other systematic immunosuppressant other than glucocorticoids within 30 days prior to enrollment.
- Patients with vaccination history within 30 days prior to screening.
- Have potential liver diseases, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy or liver fibrosis (fibrosis...
See the full eligibility criteria
- people are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent.
- Males ≥ 18 years of age.
- Have hemophilia A with ≤2 IU/dL (≤2 %) endogenous FVIII activity levels.
- Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
- Have had bleeding events and/or infusions with FVIII protein products (including recombination and plasma source) during the last 12 weeks documented in the people' medical records.
- Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration.
- Have no FVIII inhibitor. (eg \<0.6BU/ml Bethesda Units; or the patient's FVIII inhibitor titer was detected \<0.6BU/ml in 2 consecutive times within 1-4 weeks using Bethesda method or Nijmegen method), or no prior...
- Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after BBM002 infusion.
- Compliance is good, patients and their families have the will of 'gene therapy' clinical trials.
- 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).
- Currently on antiviral therapy for hepatitis B or C.
- Patients with coagulation disorders in addition to hemophilia A.
- Use of any other systematic immunosuppressant other than glucocorticoids within 30 days prior to enrollment.
- Patients with vaccination history within 30 days prior to screening.
- 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...
- Patients with known planned major surgery schedule during the 52-week study period aren't eligible.
- 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.
- Have alcohol or drug dependence, or cannot stop drinking throughout the study. 10.Any concurrent clinically significant major disease or condition that the investigator deems 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, 18 years and older. 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.