Actively Recruiting

Early Phase 1
Age: 18Years +
MALE
NCT06238908

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21

6

Participants Needed

1

Research Sites

315 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Who Can Participate

Age: 18Years +
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Voluntarily sign the informed consent form
  • Male, age 18 years old
  • Diagnosed with hemophilia A with endogenous FVIII activity level less than 1 IU/dL (less than 1%)
  • Have at least 150 exposure days of treatment with any recombinant or plasma-derived FVIII product
  • Anti-AAV neutralizing antibody titer less than or equal to 1:5 and binding antibody titer less than or equal to 1:100
  • Experienced bleeding events and/or FVIII product injections within 12 weeks before screening
  • No history of allergy to FVIII products
  • FVIII inhibitor titer less than 0.6 BU/mL
  • Agree to use other drugs during the study only with investigator consent
  • Willing and able to follow study procedures and requirements
  • Agree to use effective contraception methods within 52 weeks after treatment
Not Eligible

You will not qualify if you...

  • Positive for hepatitis B surface antigen, hepatitis C, HIV, or syphilis test
  • Abnormal liver function tests: ALT greater than 1.5 times upper limit of normal (ULN) and/or AST greater than 1.5 times ULN; total bilirubin greater than 1.5 times ULN; serum creatinine greater than 1.5 times ULN; hemoglobin less than 110 g/L; platelets less than 10e9/L
  • History of positive FVIII inhibitors
  • Have bleeding disorders other than hemophilia
  • Planning major surgery within 52 weeks
  • Contraindications to glucocorticoids including allergy, epilepsy, new unhealed fractures, trauma repair, uncontrolled infection, severe osteoporosis, as assessed by investigator
  • History of allergy to human albumin
  • Serious diseases or active infections in cardiovascular, respiratory, digestive, endocrine, kidney, blood, nervous, mental, or other systems
  • Have hepatitis, cirrhosis, liver cancer, or other major liver diseases
  • History of cancer
  • Abnormal or clinically significant vital signs, physical exam, lab or other exams unsuitable for trial as per investigator
  • Previous gene therapy treatment
  • Participated in another clinical trial recently and used medication within four weeks or five half-lives of that drug
  • Any other condition that makes participation inappropriate as judged by the investigator

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China, 300020

Actively Recruiting

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Research Team

W

Wei Liu, MD

CONTACT

L

Lei Zhang, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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