Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector （TYF-IL-2Rg）
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| ClinicalTrials.gov Identifier: NCT03217617 |
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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
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Sponsor:
Shenzhen Geno-Immune Medical Institute
Information provided by (Responsible Party):
Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute
| Tracking Information | ||||||||||
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| First Submitted Date ICMJE | July 3, 2017 | |||||||||
| First Posted Date ICMJE | July 14, 2017 | |||||||||
| Last Update Posted Date | September 19, 2019 | |||||||||
| Actual Study Start Date ICMJE | July 15, 2017 | |||||||||
| Estimated Primary Completion Date | December 31, 2019 (Final data collection date for primary outcome measure) | |||||||||
| Current Primary Outcome Measures ICMJE |
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| Original Primary Outcome Measures ICMJE |
percentage of treated patients with adverse events [ Time Frame: up to one month ] Incidents of treatment related adverse events as assessed by CTCAE V4.0
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| Change History | ||||||||||
| Current Secondary Outcome Measures ICMJE | Not Provided | |||||||||
| Original Secondary Outcome Measures ICMJE |
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| Current Other Pre-specified Outcome Measures | Not Provided | |||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | |||||||||
| Descriptive Information | ||||||||||
| Brief Title ICMJE | Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector （TYF-IL-2Rg） | |||||||||
| Official Title ICMJE | Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector （TYF-IL-2Rg） | |||||||||
| 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. | |||||||||
| 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 ICMJE | Interventional | |||||||||
| Study Phase ICMJE | Phase 1 Phase 2 |
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| Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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| Condition ICMJE | SCID, X Linked | |||||||||
| Intervention ICMJE | Biological: TYF-IL-2Rg gene-modified autologous stem cells
Infusion of transduced autologous stem cells
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| Study Arms ICMJE | Experimental: Single arm
Gene transfer to treat SCID-X1
Intervention: Biological: TYF-IL-2Rg gene-modified autologous stem cells
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| Publications * | Not Provided | |||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | ||||||||||
| Recruitment Status ICMJE | Unknown status | |||||||||
| Estimated Enrollment ICMJE |
10 | |||||||||
| Original Estimated Enrollment ICMJE | Same as current | |||||||||
| Estimated Study Completion Date ICMJE | December 31, 2020 | |||||||||
| Estimated Primary Completion Date | December 31, 2019 (Final data collection date for primary outcome measure) | |||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria: None |
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| Sex/Gender ICMJE |
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| Ages ICMJE | 1 Month to 10 Years (Child) | |||||||||
| Accepts Healthy Volunteers ICMJE | No | |||||||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||||||||
| Listed Location Countries ICMJE | China | |||||||||
| Removed Location Countries | ||||||||||
| Administrative Information | ||||||||||
| NCT Number ICMJE | NCT03217617 | |||||||||
| Other Study ID Numbers ICMJE | GIMI-IRB-17014 | |||||||||
| Has Data Monitoring Committee | No | |||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Current Responsible Party | Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute | |||||||||
| Original Responsible Party | Same as current | |||||||||
| Current Study Sponsor ICMJE | Shenzhen Geno-Immune Medical Institute | |||||||||
| Original Study Sponsor ICMJE | Same as current | |||||||||
| Collaborators ICMJE | Not Provided | |||||||||
| Investigators ICMJE |
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| PRS Account | Shenzhen Geno-Immune Medical Institute | |||||||||
| Verification Date | September 2019 | |||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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