AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

← Trials
🧪

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

RecruitingEARLY Phase 1Ages 18 Years - anyMaleInstitute of Hematology & Blood Diseases Hospital…
💬

In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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.
💊

What it tests

  • NGGT003
📍

Where

1 site
Institute of Hematology & Blood Diseases Hospital
Tianjin, China
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
🛡️

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy