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NCT03217032
Lentiviral FVIII Gene Therapy
Phase 1 trial testing YUVA-GT-F801 in Hemophilia A in 10 participants. Status unknown.
31 May 2022
Quick facts
| Lead sponsor | Shenzhen Geno-Immune Medical Institute |
|---|---|
| Phase | Phase 1 |
| Status | Status unknown |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 10 |
| Start date | 1 June 2020 |
| Primary completion | 31 May 2022 |
| Estimated completion | 1 June 2022 |
| Sites | 1 location across China |
Drugs / interventions tested
- YUVA-GT-F801 — full drug profile →
Conditions studied
- Hemophilia A — all drugs for Hemophilia A →
Sponsor
Shenzhen Geno-Immune Medical Institute
Who can join
Adults 2 to 65, any sex, with Hemophilia A. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Update on clinical gene therapy for hemophilia.
Perrin GQ, Herzog RW, Markusic DM. · · 2019 · cited 128× · PMID 30559260 · DOI 10.1182/blood-2018-07-820720 -
Novel vectors and approaches for gene therapy in liver diseases.
Maestro S, Weber ND, Zabaleta N, Aldabe R, et al · · 2021 · cited 81× · PMID 34159305 · DOI 10.1016/j.jhepr.2021.100300 -
A Molecular Revolution in the Treatment of Hemophilia.
Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. · · 2020 · cited 76× · PMID 31843450 · DOI 10.1016/j.ymthe.2019.11.006 -
Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges.
Martínez-Molina E, Chocarro-Wrona C, Martínez-Moreno D, Marchal JA, et al · · 2020 · cited 55× · PMID 33153183 · DOI 10.3390/pharmaceutics12111051 -
Recent Advances in the Treatment of Hemophilia: A Review.
Marchesini E, Morfini M, Valentino L. · · 2021 · cited 53× · PMID 34163136 · DOI 10.2147/btt.s252580 -
Gene Therapy in Hemophilia: Recent Advances.
Rodríguez-Merchán EC, De Pablo-Moreno JA, Liras A. · · 2021 · cited 32× · PMID 34299267 · DOI 10.3390/ijms22147647 -
Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective.
Tomeo F, Mariz S, Brunetta AL, Stoyanova-Beninska V, et al · · 2021 · cited 23× · PMID 33772837 · DOI 10.1111/bcp.14838 -
An overview of development in gene therapeutics in China.
Wang D, Wang K, Cai Y. · · 2020 · cited 20× · PMID 32528163 · DOI 10.1038/s41434-020-0163-7
Verify or expand the search:
- PubMed search for NCT03217032
- 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.
- NCT07416526 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A · Phase 3 · recruiting
- NCT07416604 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A · Phase 3 · recruiting
- 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 Shenzhen Geno-Immune Medical Institute trials
Trials by the same sponsor.
- NCT07501156 — H3K27M-specific Immune Effector Cells Targeting DMG/DIPG · Phase 1, PHASE2 · recruiting
- NCT07495956 — cfMSC Therapy for Diabetes · Phase 1, PHASE2 · recruiting
- NCT07477600 — cfMSC Stem Cell Therapy Targeting COPD · Phase 1, PHASE2 · recruiting
- NCT07180927 — DLL3 CAR-T Therapy Targeting Brain Tumors · Phase 1, PHASE2 · recruiting
- NCT07193966 — NG2 and DLL3 CAR-T Cells Targeting Melanoma · Phase 1, PHASE2 · recruiting
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 NCT03217032 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Shenzhen Geno-Immune Medical Institute
- Last refreshed: 23 May 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03217032.
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