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

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. Identifier: NCT03217617
Recruitment Status : Unknown
Verified September 2019 by Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute.
Recruitment status was:  Recruiting
First Posted : July 14, 2017
Last Update Posted : September 19, 2019
Information provided by (Responsible Party):
Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
SCID, X Linked Biological: TYF-IL-2Rg gene-modified autologous stem cells Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)
Actual Study Start Date : July 15, 2017
Estimated Primary Completion Date : December 31, 2019
Estimated Study Completion Date : December 31, 2020

Arm Intervention/treatment
Experimental: Single arm
Gene transfer to treat SCID-X1
Biological: TYF-IL-2Rg gene-modified autologous stem cells
Infusion of transduced autologous stem cells

Primary Outcome Measures :
  1. Overall survival [ Time Frame: 1 year ]
  2. Overall immune reconstitution [ Time Frame: 1 year ]
    T and B cell recovery

  3. Change of infection status [ Time Frame: 1 year ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Month to 10 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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:


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 identifier (NCT number): NCT03217617

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Contact: Lung-Ji Chang, Ph.D 86-075586725195

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China, Beijing
Capital Institute of Pediatrics affiliated Children's hospital Recruiting
Beijing, Beijing, China, 100020
Contact: XiaoDong Shi, M.D./P.H.D    +86-13911601076   
Contact: Lixiao Shi, M.M.    +86-18810963129   
Beijing Children's Hospital Recruiting
Beijing, Beijing, China
Contact: Jie Zheng, MD/PhD    +86-13683284467   
China, Guangdong
Shenzhen Geno-immune Medical Institute Recruiting
Shenzhen, Guangdong, China, 518000
Contact: Lung-Ji Chang, PhD    86-075586725195   
Sponsors and Collaborators
Shenzhen Geno-Immune Medical Institute
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Principal Investigator: Lung-Ji Chang, Ph.D Shenzhen Geno-Immune Medical Institute
Study Director: Xiao-Dong Shi, M.D./Ph. D Capital Institute of Pediatrics affiliated Children's hospital
Study Director: Jie Zheng, M.D./Ph. D Beijing Children's Hospital
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Responsible Party: Lung-Ji Chang, President, Shenzhen Geno-Immune Medical Institute Identifier: NCT03217617    
Other Study ID Numbers: GIMI-IRB-17014
First Posted: July 14, 2017    Key Record Dates
Last Update Posted: September 19, 2019
Last Verified: September 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 Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute:
lentiviral vector
Additional relevant MeSH terms:
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X-Linked Combined Immunodeficiency Diseases
Severe Combined Immunodeficiency
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Genetic Diseases, X-Linked