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Lentiviral FVIII Gene Therapy

Primary Purpose

Hemophilia A

Status
Unknown status
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
YUVA-GT-F801
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 A focused on measuring Hemophilia A, factor VIII, Gene therapy, lentiviral 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 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.

Sites / Locations

  • Shenzhen Geno-immune Medical Institute

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

YUVA-GT-F801

Arm Description

Gene transfer to treat Hemophilia A

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 FVIII inhibitor

Secondary Outcome Measures

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

Full Information

First Posted
July 3, 2017
Last Updated
May 21, 2019
Sponsor
Shenzhen Geno-Immune Medical Institute
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1. Study Identification

Unique Protocol Identification Number
NCT03217032
Brief Title
Lentiviral FVIII Gene Therapy
Official Title
Lentiviral FVIII Gene Therapy for Hemophilia A
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
Principal Investigator
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 VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
Detailed Description
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
Hemophilia A, factor VIII, Gene therapy, lentiviral 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-F801
Arm Type
Experimental
Arm Description
Gene transfer to treat Hemophilia A
Intervention Type
Biological
Intervention Name(s)
YUVA-GT-F801
Intervention Description
Lentiviral factor VIII gene modified autologous hematopoeitic stem cells 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 FVIII inhibitor
Time Frame
a year
Secondary Outcome Measure Information:
Title
Changes from baseline in circulating FVIII 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: Able to provide informed consent and comply with requirements of the study. Males ≥2 years with confirmed diagnosis of hemophilia A (endogenous factor VIII ≤2 IU/dL or ≤2% of normal). A minimum average of 4 bleeding events per year requiring episodic treatment of factor VIII infusions or prophylactic factor VIII infusions. No measurable factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor VIII protein. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences. Exclusion Criteria: Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase. History of inhibitor against factor VIII. Evidence of active hepatitis B or C and currently on antiviral therapy. 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). Any evidence of active infection or any immunosuppressive disorder. 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. 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
Organizational Affiliation
Shenzhen Geno-Immune Medical Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shenzhen Geno-immune Medical Institute
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518000
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No

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Lentiviral FVIII Gene Therapy

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