NCT06238908 Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
| NCT ID | NCT06238908 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China |
| Condition | Hemophilia A |
| Study Type | INTERVENTIONAL |
| Enrollment | 6 participants |
| Start Date | 2024-01-17 |
| Primary Completion | 2026-01-31 |
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 6 participants in total. It began in 2024-01-17 with a primary completion date of 2026-01-31.
⚠ 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 an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. NGGT003 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant (hFVIII BDD), and expresses human FVIII protein in the liver through intravenous injection.
Eligibility Criteria
Inclusion Criteria: 1. Voluntarily sign the informed consent form; 2. Male, age ≥18 years old; 3. Diagnosed with hemophilia A according to the "Guidelines for Diagnosis and Treatment of Hemophilia A (2022 Edition)", and the endogenous FVIII activity level was \<1 IU/dL (\<1%); 4. The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days; 5. Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100; 6. Bleeding events and/or FVIII product injections have occurred within 12 weeks before screening; 7. No history of allergy to FVIII products; 8. FVIII inhibitor titer﹤0.6BU/mL; 9. Commitment to use other drugs during the study requires the consent of the investigator; 10. Willing and able to comply with study procedures and requirements; 11. Willing to use effective contraceptive methods within 52 weeks after administration. Exclusion Criteria: 1. Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test; 2. Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) \>1.5 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) \>1.5× ULN;TBil）\>1.5×ULN；Serum creatinine (Scr) \>1.5×ULN; hemoglobin \<110g/L, platelets \<10e9/L; 3. History of being positive for FVIII inhibitors; 4. Have other bleeding factors except hemophilia; 5. Plan major surgery within 52 weeks; 6. Have contraindications to glucocorticoid, including but not limited to allergy to glucocorticoids, epilepsy, new unhealed fractures, in trauma repair period, uncontrolled infection, severe osteoporosis, etc, which assessed and determined by the investigators; 7. History of allergy to human albumin; 8. Have serious diseases or active infections in cardiovascular, respiratory, digestive tract, endocrine, renal, blood, nervous, mental and other systems before screening; 9. With hepatitis, cirrhosis, liver cancer or other major liver diseases; 10. History of malignant tumors; 11. Abnormal and clinical significant vital signs, physical examination, laboratory examination or other related examination results during the screen, which are not suitable for trial according to the investigator; 12. Previous gene therapy treatment; 13. Participation in any other clinical trial before the screening and have taken medication within four weeks or five half-lives of the study drug; 14. Any other condition that may not be appropriate for the study in the opinion of the investigator.
Contact & Investigator
Lei Zhang, MD
PRINCIPAL INVESTIGATOR
Institute of Hematology & Blood Diseases Hospital, China
Frequently Asked Questions
Who can join the NCT06238908 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 NCT06238908 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 NCT06238908 currently recruiting?
Yes, NCT06238908 is actively recruiting participants. Contact the research team at liuwei1@ihcams.ac.cn for enrollment information.
Where is the NCT06238908 trial being conducted?
This trial is being conducted at Tianjin, China.
Who is sponsoring the NCT06238908 clinical trial?
NCT06238908 is sponsored by Institute of Hematology & Blood Diseases Hospital, China. The principal investigator is Lei Zhang, MD at Institute of Hematology & Blood Diseases Hospital, China. The trial plans to enroll 6 participants.