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NCT06238908

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Recruiting now EARLY_PHASE1 Last updated 21 February 2025
What this trial tests

EARLY_PHASE1 trial testing NGGT003 in Hemophilia A in 6 participants. Currently enrolling.

Timeline
17 January 2024
Primary endpoint
31 January 2026
31 January 2030

Quick facts

Lead sponsorInstitute of Hematology & Blood Diseases Hospital, China
PhaseEARLY_PHASE1
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment6
Start date17 January 2024
Primary completion31 January 2026
Estimated completion31 January 2030
Sites1 location across China

Drugs / interventions tested

Conditions studied

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Who can join

18 and older, male only, with Hemophilia A. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

Verify or expand the search:

Other recruiting trials for Hemophilia A

Currently open trials in the same condition.

Other Institute of Hematology & Blood Diseases Hospital, China trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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