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Tests treatment safety and results for Hemophilia A
Official title Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
ClinicalTrials.gov ID: NCT06238908
What this study is testing
What is NGGT003?
NGGT003 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia a.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older, men only
You may be able to join if
- Voluntarily sign the informed consent form;
- Male, age ≥18 years old;
- Diagnosed with hemophilia A according to the "Guidelines for Diagnosis and Treatment of Hemophilia A (2022 Edition)", and the endogenous FVIII...
- The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days;
- Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100;
You likely can't join if
- Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test;
- Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) \>1.5 × upper limit of normal (ULN) and/or aspartate...
- History of being positive for FVIII inhibitors;
- Have other bleeding factors except hemophilia;
- Plan major surgery within 52 weeks;
- Have contraindications to glucocorticoid, including but not limited to allergy to glucocorticoids, epilepsy, new unhealed fractures, in trauma repair...
See the full eligibility criteria
- Voluntarily sign the informed consent form;
- Male, age ≥18 years old;
- 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%);
- The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days;
- Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100;
- Bleeding events and/or FVIII product injections have occurred within 12 weeks before screening;
- No history of allergy to FVIII products;
- FVIII inhibitor titer﹤0.6BU/mL;
- Commitment to use other drugs during the study requires the consent of the investigator;
- Willing and able to comply with study procedures and requirements;
- Willing to use effective contraceptive methods within 52 weeks after administration.
- Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test;
- 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)...
- History of being positive for FVIII inhibitors;
- Have other bleeding factors except hemophilia;
- Plan major surgery within 52 weeks;
- 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...
- History of allergy to human albumin;
- Have serious diseases or active infections in cardiovascular, respiratory, digestive tract, endocrine, renal, blood, nervous, mental and other systems before screening;
- With hepatitis, cirrhosis, liver cancer or other major liver diseases;
- History of malignant tumors;
- 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;
- Previous gene therapy treatment;
- Participation in any other clinical trial before the screening and have taken medication within four weeks or five half-lives of the study drug;
- Any other condition that may not be appropriate for the study in the opinion of the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Tianjin, Tianjin Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Tianjin, Tianjin Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.