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Not yet recruitingNCT03217032Updated Sep 1, 2026

Lentiviral FVIII Gene Therapy

A Phase 1 interventional study of YUVA-GT-F801 in Hemophilia A, sponsored by Shenzhen Geno-Immune Medical Institute. Not yet recruiting at 1 site in China. Open to participants aged 2 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-09-01.

Sponsored by Shenzhen Geno-Immune Medical Institute · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
2 Years to 65 Years
Sex
All
01

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.

Read the detailed description

Important Regulatory Notice:

This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China.

ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities.

Hemophilia A is a genetic bleeding disorder caused by the lack of ability to produce blood-clotting factor VIII (FVIII). Individuals with hemophilia A suffer repeated bleeding episodes, which can cause chronic joint disease and sometimes even death due to the inability for blood to clot efficiently. The current treatment is intravenous infusion of clotting factor concentrates, either prophylactically or in response to bleeding. The procedure is life time long and expensive while still cannot achieve a cure.Gene therapy is a novel technology that has been successfully demonstrated in a number of clinical studies for diseases such as cancer and genetic diseases. In this study, an advanced lentiviral vector system NHP/TYF will be used to deliver a functional FVIII gene to overcome human clotting FVIII gene defect in patients with hemophilia A. This study is a Phase I trial evaluating the safety and efficacy for infusion of gene modified autologous stem cells in patients with hemophilia A.

02

Conditions studied

  • Hemophilia A

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Keywords

  • Hemophilia A
  • factor VIII
  • Gene therapy
  • lentiviral vector
03

Who can participate

Ages eligible
2 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (estimated)

Study arms

  • Experimental
    YUVA-GT-F801

    Gene transfer to treat Hemophilia A

    Biological: YUVA-GT-F801

Interventions

  • BiologicalYUVA-GT-F801

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

05

What researchers measure

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

06

Study locations

1 site
  • Shenzhen Geno-immune Medical Institute
    Shenzhen, Guangdong 518000, China
    • Lung-Ji Chang, PhD · Contact · c@szgimi.org · +86 0755-86573763
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03217032
Lead sponsor
Shenzhen Geno-Immune Medical Institute
Responsible party
Lung-Ji Chang (President, Shenzhen Geno-Immune Medical Institute) — Principal investigator
First posted
Jul 13, 2017
Start date
Jun 1, 2027 (estimated)
Primary completion
May 31, 2028 (estimated)
Completion
Dec 1, 2028 (estimated)
Last update
Sep 1, 2026

Study contacts

Lung-Ji Chang, PhD
Contact
c@szgimi.org
+86 0755-86573763
Lung-Ji Chang
principal investigator · Shenzhen Geno-Immune Medical Institute

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.

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