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Lentiviral FIX Gene Therapy

Primary Purpose

Hemophilia B

Status
Unknown status
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
YUVA-GT-F901
Sponsored by
Shenzhen Geno-Immune Medical Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia B focused on measuring Hemophilia B, factor IX, Gene therapylentiviral vector

Eligibility Criteria

2 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    YUVA-GT-F901

    Arm Description

    Gene transfer to treat Hemophilia B

    Outcomes

    Primary Outcome Measures

    Number of participants experiencing drug-related adverse events
    As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FIX inhibitor

    Secondary Outcome Measures

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

    Full Information

    First Posted
    May 22, 2019
    Last Updated
    May 22, 2019
    Sponsor
    Shenzhen Geno-Immune Medical Institute
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03961243
    Brief Title
    Lentiviral FIX Gene Therapy
    Official Title
    Lentiviral FIX Gene Therapy for Hemophilia B
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    May 2019
    Overall Recruitment Status
    Unknown status
    Study Start Date
    June 1, 2020 (Anticipated)
    Primary Completion Date
    May 31, 2022 (Anticipated)
    Study Completion Date
    June 1, 2022 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Shenzhen Geno-Immune Medical Institute

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    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.
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Hemophilia B
    Keywords
    Hemophilia B, factor IX, Gene therapylentiviral vector

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    10 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    YUVA-GT-F901
    Arm Type
    Experimental
    Arm Description
    Gene transfer to treat Hemophilia B
    Intervention Type
    Biological
    Intervention Name(s)
    YUVA-GT-F901
    Intervention Description
    Lentiviral factor IX gene modified autologous hematopoietic and mesenchymal stem cells
    Primary Outcome Measure Information:
    Title
    Number of participants experiencing drug-related adverse events
    Description
    As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FIX inhibitor
    Time Frame
    a year
    Secondary Outcome Measure Information:
    Title
    Changes from baseline in circulating FIX activity (IU/dL or % normal)
    Time Frame
    a year

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Lung-Ji Chang, PhD
    Phone
    86-075586725195
    Email
    c@szgimi.org
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Lung-Ji Chang, PhD
    Organizational Affiliation
    Shenzhen Geno-Immune Medical Institute
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No

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    Lentiviral FIX Gene Therapy

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