Health TrialsbyHealthPanel

Lentiviral FVIII Gene Therapy

ConditionHemophilia A
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age2-65
SponsorShenzhen Geno-Immune Medical Institute

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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).

Trial design

Design model

Single group

Treatments tested in this trial

  • YUVA-GT-F801

    Biological/Vaccine

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

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: YUVA-GT-F801Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of participants experiencing drug-related adverse events

As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FVIII inhibitor

Time frame
a year

Secondary outcomes

1

Changes from baseline in circulating FVIII activity (IU/dL or % normal)

Time frame
a year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shenzhen Geno-Immune Medical Institute

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: