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NCT06238908
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
EARLY_PHASE1 trial testing NGGT003 in Hemophilia A in 6 participants. Currently enrolling.
31 January 2026
Quick facts
| Lead sponsor | Institute of Hematology & Blood Diseases Hospital, China |
|---|---|
| Phase | EARLY_PHASE1 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 6 |
| Start date | 17 January 2024 |
| Primary completion | 31 January 2026 |
| Estimated completion | 31 January 2030 |
| Sites | 1 location across China |
Drugs / interventions tested
- NGGT003 — full drug profile →
Conditions studied
- Hemophilia A — all drugs for Hemophilia A →
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:
- PubMed search for NCT06238908
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Hemophilia A
Currently open trials in the same condition.
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- NCT07523399 — Joint Health, Balance and Quality of Life in Adults With Hemophilia A · recruiting
- NCT06833983 — To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A · Phase 3 · recruiting
- NCT06579144 — Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A · Phase 1 · recruiting
Other Institute of Hematology & Blood Diseases Hospital, China trials
Trials by the same sponsor.
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- NCT07407140 — VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML · Phase 3 · not yet recruiting
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06238908 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Institute of Hematology & Blood Diseases Hospital, China
- Last refreshed: 21 February 2025
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