RecruitingPhase 1NCT03217032

Lentiviral FVIII Gene Therapy

Lentiviral FVIII Gene Therapy for Hemophilia A

Sponsor

Shenzhen Geno-Immune Medical Institute

Enrollment

10 participants

Start Date

Jun 1, 2025

Study Type

INTERVENTIONAL

Conditions

Hemophilia A

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.

Eligibility

Min Age: 2 YearsMax Age: 65 Years

Inclusion Criteria5

Able to provide informed consent and comply with requirements of the study.
Males ≥2 years with confirmed diagnosis of hemophilia A (endogenous factor VIII ≤2 IU/dL or ≤2% of normal).
A minimum average of 4 bleeding events per year requiring episodic treatment of factor VIII infusions or prophylactic factor VIII infusions.
No measurable factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor VIII protein.
Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.

Exclusion Criteria7

Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
History of inhibitor against factor VIII.
Evidence of active hepatitis B or C and currently on antiviral therapy.
Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (subjects who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll).
Any evidence of active infection or any immunosuppressive disorder.
Participated in a gene transfer trial within the last 6 months or in a clinical trial with an investigational drug within the last 12 weeks.
Unable or unwilling to comply with study assessments.

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Interventions

BIOLOGICALYUVA-GT-F801

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

Locations(1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, China

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NCT03217032

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