NCT05454774 A Study of FVIII Gene Therapy for Hemophilia A
| NCT ID | NCT05454774 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China |
| Condition | Hemophilia A |
| Study Type | INTERVENTIONAL |
| Enrollment | 8 participants |
| Start Date | 2022-07-19 |
| Primary Completion | 2024-12-15 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 8 participants in total. It began in 2022-07-19 with a primary completion date of 2024-12-15.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 1. Subjects are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent. 2. Males ≥ 18 years of age. 3. Have hemophilia A with ≤2 IU/dL (≤2 %) endogenous FVIII activity levels. 4. Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products. 5. Have had bleeding events and/or infusions with FVIII protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records. 6. Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration. 7. 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 medical history of FVIII inhibitor after 150 EDs of FVIII products; no clinical signs or symptoms of decreased response to FVIII products infusion. 8. Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after BBM002 infusion. 9. Compliance is good, patients and their families have the will of 'gene therapy' clinical trials. 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). 2. Currently on antiviral therapy for hepatitis B or C. 3. Patients with coagulation disorders in addition to hemophilia A. 4. Use of any other systematic immunosuppressant other than glucocorticoids within 30 days prior to enrollment. 5. Patients with vaccination history within 30 days prior to screening. 6. 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 by researchers. 7. Patients with known planned major surgery schedule during the 52-week study period aren't eligible. 8. 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. 9. 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.
Contact & Investigator
Lei Zhang, MD
STUDY CHAIR
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Frequently Asked Questions
Who can join the NCT05454774 clinical trial?
This trial is open to male participants only, aged 18 Years or older, studying Hemophilia A. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05454774 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05454774 currently recruiting?
Yes, NCT05454774 is actively recruiting participants. Contact the research team at zhanglei1@ihcams.ac.cn for enrollment information.
Where is the NCT05454774 trial being conducted?
This trial is being conducted at Tianjin, China.
Who is sponsoring the NCT05454774 clinical trial?
NCT05454774 is sponsored by Institute of Hematology & Blood Diseases Hospital, China. The principal investigator is Lei Zhang, MD at Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College. The trial plans to enroll 8 participants.