Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)

September 18, 2019 updated by: Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute

Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)

This is a Phase I/II clinical trial of gene transfer for treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector TYF-IL-2Rg to functionally correct the defective gene(s). The primary objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Study Overview

Status

Unknown

Conditions

Detailed Description

X-linked severe combined immunodeficiency (SCID-X1) is a genetic disorder caused by defects in the common cytokine receptor chain, normally on the surface of lymphocytes. Individuals with SCID-X1 lack the normal development of a functional immune system and so have difficulty fighting infections, which may lead to chronic or severe illness and death. X-SCID patients are normally rescued by a bone marrow transplant from a healthy donor. This trial aims to treat SCID-X1 using a self-inactivating lentiviral vector carrying a functional gene to correct the genetic defect. By collecting an individual's stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned into the blood to help produce normal healthy immune cells.

The primary objectives are to evaluate the safety of the self-inactivating lentiviral vector TYF-IL-2Rg, the ex vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of integration sites, and finally the long-term correction of immunodeficiency.

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Lung-Ji Chang, Ph.D
  • Phone Number: 86-075586725195
  • Email: c@szgimi.org

Study Locations

    • Beijing
      • Beijing, Beijing, China, 100020
        • Recruiting
        • Capital Institute of Pediatrics affiliated Children's hospital
      • Beijing, Beijing, China
        • Recruiting
        • Beijing Children's Hospital
    • Guangdong
      • Shenzhen, Guangdong, China, 518000
        • Recruiting
        • Shenzhen Geno-Immune Medical Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month to 10 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. Diagnosis of classical SCID-X1 based on:

    • A proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA.
    • T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells < 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigens.
  2. With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection.
  3. No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children.
  4. No prior allogeneic stem cell transplantation.
  5. Life expectancy ≥ 2 months.
  6. Negative for HIV infection.
  7. Written, informed consent obtained prior to any study-specific procedures.

Exclusion Criteria:

None

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single arm
Gene transfer to treat SCID-X1
Infusion of transduced autologous stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall survival
Time Frame: 1 year
1 year
Overall immune reconstitution
Time Frame: 1 year
T and B cell recovery
1 year
Change of infection status
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 15, 2017

Primary Completion (Anticipated)

December 31, 2019

Study Completion (Anticipated)

December 31, 2020

Study Registration Dates

First Submitted

July 3, 2017

First Submitted That Met QC Criteria

July 11, 2017

First Posted (Actual)

July 14, 2017

Study Record Updates

Last Update Posted (Actual)

September 19, 2019

Last Update Submitted That Met QC Criteria

September 18, 2019

Last Verified

September 1, 2019

More Information

Terms related to this study

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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