Lentiviral FVIII Gene Therapy

Plain-language summary

This Phase 1 trial uses an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A. The patient's own stem cells are genetically modified with the vector and infused back, and the safety and efficacy of this approach are being evaluated. Enrollment is open to males aged 2 years and older with confirmed hemophilia A (endogenous factor VIII 2 IU/dL or below, i.e. 2% of normal or less) who have at least 4 bleeding events per year requiring treatment.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Recruiting

Sponsor

Shenzhen Geno-Immune Medical Institute

Intervention

YUVA-GT-F801

Start date

6/1/2025

Estimated completion

6/1/2028

Countries

China

Related genes

Locations

• Shenzhen, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

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

Exclusion Criteria:

1. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
2. History of inhibitor against factor VIII.
3. Evidence of active hepatitis B or C and currently on antiviral therapy.
4. 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).
5. Any evidence of active infection or any immunosuppressive disorder.
6. 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.
7. Unable or unwilling to comply with study assessments.