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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT03217617 |
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Date of registration:
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03/07/2017 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)
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Scientific title:
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Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) |
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Date of first enrolment:
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July 15, 2017 |
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Target sample size:
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10 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT03217617 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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China
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Contacts
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Name:
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Lung-Ji Chang, Ph.D |
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Address:
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Telephone:
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Email:
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Affiliation:
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Shenzhen Geno-Immune Medical Institute |
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Name:
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Jie Zheng, M.D./Ph. D |
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Address:
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Telephone:
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Email:
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Affiliation:
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Beijing Children's Hospital |
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Name:
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Lung-Ji Chang, Ph.D |
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Address:
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Telephone:
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86-075586725195 |
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Email:
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c@szgimi.org |
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Affiliation:
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Name:
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Xiao-Dong Shi, M.D./Ph. D |
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Address:
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Telephone:
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Email:
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Affiliation:
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Capital Institute of Pediatrics affiliated Children's hospital |
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Key inclusion & exclusion criteria
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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
Age minimum:
1 Month
Age maximum:
10 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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SCID, X Linked
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Intervention(s)
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Biological: TYF-IL-2Rg gene-modified autologous stem cells
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Primary Outcome(s)
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Overall survival
[Time Frame: 1 year]
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Overall immune reconstitution
[Time Frame: 1 year]
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Change of infection status
[Time Frame: 1 year]
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Secondary ID(s)
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GIMI-IRB-17014
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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