Evaluating NGGT003 for treating Hemophilia A

Clinical Study on the Safety and Efficacy of an Intravenous Infusion of NGGT003 in the Treatment of Hemophilia A

This study is testing a new treatment called NGGT003 to see if it can safely help men with hemophilia A by allowing their bodies to produce a missing protein.

Quick facts

• Phase: EARLY_PHASE1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 18 Years and up
• Sex: Male
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (other)
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT06238908 on ClinicalTrials.gov

What this trial studies

This early phase 1, open-label, single-center pilot trial aims to assess the safety and efficacy of NGGT003, an intravenous infusion designed for hemophilia A patients. The study utilizes an adeno-associated virus (AAV) vector to deliver a modified human FVIII gene to the liver, potentially enabling the production of the FVIII protein. A total of 4-6 male participants aged 18 and older will be enrolled, receiving escalating doses of NGGT003, with a follow-up period extending up to 260 weeks to monitor long-term effects.

Who should consider this trial

Good fit: Ideal candidates are adult males diagnosed with hemophilia A who have low endogenous FVIII activity and a history of FVIII product treatment.

Not a fit: Patients with a history of hepatitis B, hepatitis C, or HIV, or those with high FVIII inhibitor titers may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve coagulation function and reduce bleeding episodes in hemophilia A patients.

How similar studies have performed: While gene therapy approaches for hemophilia are being explored, this specific method using NGGT003 is novel and has not been extensively tested in prior studies.

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.

Where this trial is running

Tianjin, Tianjin Municipality

• Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin Municipality, China (RECRUITING)

Study contacts

• Principal investigator: Lei Zhang, MD — Institute of Hematology & Blood Diseases Hospital, China
• Study coordinator: Wei Liu, MD

Conditions: Hemophilia A