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Gene Therapy for X Linked Severe Combined Immunodeficiency

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04286815
Recruitment Status : Recruiting
First Posted : February 27, 2020
Last Update Posted : March 27, 2020
Sponsor:
Information provided by (Responsible Party):
mingfeng hu, Children's Hospital of Chongqing Medical University

Brief Summary:
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID(severe combined immune deficiency ).The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.

Condition or disease Intervention/treatment Phase
Gene Therapy Device: Lentiviral Vector Gene Therapy Not Applicable

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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 Therapy for X Linked Severe Combined Immunodeficiency
Estimated Study Start Date : May 1, 2020
Estimated Primary Completion Date : May 1, 2023
Estimated Study Completion Date : May 1, 2025


Arm Intervention/treatment
Experimental: Experimental Group
a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID(severe combined immune deficiency).
Device: Lentiviral Vector Gene Therapy
Lentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells




Primary Outcome Measures :
  1. 1-year survival rate 1-year survival rate [ Time Frame: one year after gene therapy of last recruited patient ]
    1-year survival rate of 10 recruited patients

  2. 3-year survival rate [ Time Frame: three years after gene therapy of last recruited patient ]
    3-year survival rate of 10 recruited patients

  3. 5-year survival rate [ Time Frame: five years after gene therapy of last recruited patient ]
    5-year survival rate of 10 recruited patients


Secondary Outcome Measures :
  1. Growth velocity after gene therapy,weight in kilograms, height in meters [ Time Frame: through study completion, an average of 2 year ]
    Body weight and height of patients will be assessed prior to (month 0) and post gene therapy,weight in kilograms, height in meters

  2. Vector marking (vector copy number per cell) in blood and bone marrow cells [ Time Frame: through study completion, an average of 1 year ]
    vector marking in T cells, B cells, NK cells, myeloid cells, and bone marrow progenitors.

  3. Absolute numbers of peripheral-blood immune-cell subsets [ Time Frame: through study completion, an average of 1 year ]
    Absolute numbers of peripheral-blood immune-cell subsets,as determined by means of standard flow cytometry

  4. Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood mononuclear cells [ Time Frame: through study completion, an average of 1 year ]
    Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood ,as determined by means of quantitative polymerase chain reaction (PCR)

  5. Serum immunoglobulins levels [ Time Frame: through study completion, an average of 2 year ]
    Serum immunoglobulins levels will be reported IgM(immunoglobulin M) in mg/dL Serum immunoglobulins levels will be reported IgM in mg/dL

  6. Number of patients without intravenous immune globulin supplementation [ Time Frame: through study completion, an average of 2 year ]
    Number of patients without intravenous immune globulin supplementation after gene therapy

  7. Number of patients who has a response to vaccines [ Time Frame: through study completion, an average of 2 year ]
    Number of patients who has a response to vaccines after gene therapy

  8. Number of patients who recovers from previous infection(virus and bacteria) [ Time Frame: through study completion, an average of 2 year ]
    Number of patients who recovers from previous infection(virus and bacteria)after gene therapy



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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. X-SCID patients diagnosed by IL2RG single gene mutation
  2. No HLA(human leukocyte antigen) matching donor
  3. Hematopoietic stem cell transplantation failed and the time from transplantation was more than 18 months
  4. Severe and persistent refractory infections
  5. Life expectancy of > : 4 months
  6. HIV PCR in peripheral blood was negative
  7. the children and their families signed informed consent and were willing to enter the clinical trial and complete follow-up

Exclusion Criteria:

  1. The patient has diagnosed with hematological malignant diseases
  2. Received chemotherapy within 3 months
  3. HIV infection or HBV(hepatitis B virus) infection
  4. The patient or his first-degree relative has developed a malignant tumor within the age of 18 or has been diagnosed with malignant tumor prone genes
  5. Although the patient with X-SCID was diagnosed as IL2RG single gene mutation , the clinical phenotype was not severe, so they could continue to wait for the donor search;
  6. Patients whose family members have no intention to continue the follow-up treatment in any link

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): NCT04286815


Contacts
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Contact: Xiaodong Zhao, PHD 18623070626 zhaoxd530@aliyun.com
Contact: Qiling Xu, MD 18581059910 272864835@qq.com

Locations
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China, Chongqing
Children's Hospital of Chongqing Medical University Recruiting
Chongqing, Chongqing, China, 400014
Contact: Xiaodong Zhao, PhD    18623070626    zhaoxd530@aliyun.com   
Sponsors and Collaborators
Children's Hospital of Chongqing Medical University
Investigators
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Study Director: Xiaodong Zhao, PHD Assistant President of Children's Hospital of Chongqing Medical University
Publications:
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Responsible Party: mingfeng hu, Resident physician, Children's Hospital of Chongqing Medical University
ClinicalTrials.gov Identifier: NCT04286815    
Other Study ID Numbers: CHCMU gene therapy
First Posted: February 27, 2020    Key Record Dates
Last Update Posted: March 27, 2020
Last Verified: March 2020
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 mingfeng hu, Children's Hospital of Chongqing Medical University:
Gene Therapy
X Linked Severe Combined Immunodeficiency
Lentiviral Vector
Additional relevant MeSH terms:
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Severe Combined Immunodeficiency
X-Linked Combined Immunodeficiency Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn