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Lentiviral FIX Gene Therapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03961243
Recruitment Status : Unknown
Verified May 2019 by Shenzhen Geno-Immune Medical Institute.
Recruitment status was:  Not yet recruiting
First Posted : May 23, 2019
Last Update Posted : May 23, 2019
Sponsor:
Information provided by (Responsible Party):
Shenzhen Geno-Immune Medical Institute

Study Description
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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.

Condition or disease Intervention/treatment Phase
Hemophilia B Biological: YUVA-GT-F901 Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Lentiviral FIX Gene Therapy for Hemophilia B
Estimated Study Start Date : June 1, 2020
Estimated Primary Completion Date : May 31, 2022
Estimated Study Completion Date : June 1, 2022

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Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants experiencing drug-related adverse events [ Time Frame: a year ]
    As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FIX inhibitor


Secondary Outcome Measures :
  1. Changes from baseline in circulating FIX activity (IU/dL or % normal) [ Time Frame: a year ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03961243


Contacts
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Contact: Lung-Ji Chang, PhD 86-075586725195 c@szgimi.org

Sponsors and Collaborators
Shenzhen Geno-Immune Medical Institute
Investigators
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Principal Investigator: Lung-Ji Chang, PhD Shenzhen Geno-Immune Medical Institute
More Information
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Responsible Party: Shenzhen Geno-Immune Medical Institute
ClinicalTrials.gov Identifier: NCT03961243    
Other Study ID Numbers: GIMI-IRB-19002
First Posted: May 23, 2019    Key Record Dates
Last Update Posted: May 23, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Shenzhen Geno-Immune Medical Institute:
Hemophilia B
factor IX
Gene therapylentiviral vector
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked