Lentiviral FIX Gene Therapy

Lentiviral FIX Gene Therapy for Hemophilia B

This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

Study Overview

• Status: Recruiting
• Conditions: Hemophilia B
• Intervention / Treatment: Biological: YUVA-GT-F901

Detailed Description

Hemophilia B is a genetic bleeding disorder caused by the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B 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 FIX gene to overcome human clotting FIX gene defect in patients with hemophilia B. This study is a Phase I trial evaluating the safety and efficacy for infusion of gene modified autologous stem cells in patients with hemophilia B.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Lung-Ji Chang, PhD; Phone Number: +86 0755-86573763; Email: c@szgimi.org

Study Locations

• China
  • Guangdong
    • Shenzhen, Guangdong, China, 518000
      • Recruiting
      • Shenzhen Geno-immune Medical Institute
      • Contact: Lung-Ji Chang, Ph.D; Phone Number: +86 0755-86573763; Email: c@szgimi.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 1. Able to provide informed consent and comply with requirements of the study. 2. Males ≥2 years with confirmed diagnosis of hemophilia B (endogenous factor IX ≤2 IU/dL or ≤2% of normal).

  3. A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions.

  4. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX 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 IX. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: YUVA-GT-F901; Gene transfer to treat Hemophilia B	Biological: YUVA-GT-F901; Lentiviral factor IX gene modified autologous hematopoietic and mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants experiencing drug-related adverse events	As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FIX inhibitor	a year

Secondary Outcome Measures

Outcome Measure	Time Frame
Changes from baseline in circulating FIX activity (IU/dL or % normal)	a year

Collaborators and Investigators

• Sponsor: Shenzhen Geno-Immune Medical Institute
• Investigators: Principal Investigator: Lung-Ji Chang, PhD, Shenzhen Geno-immune Medical Institute

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2025
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): June 1, 2028

Study Registration Dates

• First Submitted: May 22, 2019
• First Submitted That Met QC Criteria: May 22, 2019
• First Posted (Actual): May 23, 2019

Study Record Updates

• Last Update Posted (Actual): April 24, 2026
• Last Update Submitted That Met QC Criteria: April 21, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Hemophilia B; factor IX; Gene therapylentiviral vector
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia B
• Other Study ID Numbers: GIMI-IRB-19002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No