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Gene Therapy Trial Report

Summary

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients


NCTID NCT06238908 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name NGGT003
Sponsor Institute of Hematology & Blood Diseases Hospital, China
Funder Type Other
Recruitment Status
Enrollment Count 6 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant F8
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 4E11 vg/kg
Dose 2 1E12 vg/kg
Dose 3 2.5E12 vg/kg

Study Record Dates


Current Stage Early phase1
Submit Date 2024-01-26
Completion Date 2030-01-31
Last Update 2025-02-21

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links