Lentiviral FVIII Gene Therapy

NCT03217032 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-04-24

No results posted yet for this study

Summary

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.

Conditions

Interventions

BIOLOGICAL

YUVA-GT-F801

Lentiviral factor VIII gene modified autologous hematopoeitic stem cells and mesenchymal stem cells

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

Principal Investigators

  • Lung-Ji Chang · Shenzhen Geno-Immune Medical Institute

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-01
Primary Completion
2027-05-31
Completion
2028-06-01

Countries

  • China

Study Locations

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Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03217032 on ClinicalTrials.gov