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NCT03217032

Lentiviral FVIII Gene Therapy

Status unknown Phase 1 Last updated 23 May 2019
What this trial tests

Phase 1 trial testing YUVA-GT-F801 in Hemophilia A in 10 participants. Status unknown.

Timeline
1 June 2020
Primary endpoint
31 May 2022
1 June 2022

Quick facts

Lead sponsorShenzhen Geno-Immune Medical Institute
PhasePhase 1
StatusStatus unknown
Study typeINTERVENTIONAL
Allocationnon randomized
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment10
Start date1 June 2020
Primary completion31 May 2022
Estimated completion1 June 2022
Sites1 location across China

Drugs / interventions tested

Conditions studied

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):

  1. 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
  2. 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
  3. 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
  4. 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
  5. 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
  6. Gene Therapy in Hemophilia: Recent Advances.
    Rodríguez-Merchán EC, De Pablo-Moreno JA, Liras A. · · 2021 · cited 32× · PMID 34299267 · DOI 10.3390/ijms22147647
  7. 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
  8. 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:

Other recruiting trials for Hemophilia A

Currently open trials in the same condition.

Other Shenzhen Geno-Immune Medical Institute trials

Trials by the same sponsor.

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