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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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29 July 2024 |
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Main ID: |
NCT03645460 |
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Date of registration:
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17/07/2018 |
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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 Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
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Scientific title:
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Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID) |
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Date of first enrolment:
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June 30, 2024 |
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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/ct2/show/NCT03645460 |
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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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N/A
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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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Lung-Ji Chang, Ph.D |
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Address:
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Telephone:
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86-0755-86725195 |
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Email:
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c@szgimi.org |
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Diagnosis of classical ADA-SCID based on:
- A proven defective adenosine deaminase (ADA) 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.
- 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.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main
rearrangements associated with acute leukemia of children.
- No prior allogeneic stem cell transplantation.
- Life expectancy = 2 months.
- Negative for HIV infection.
- Written, informed consent obtained prior to any study-specific procedures.
Exclusion Criteria:
- None
Age minimum:
1 Month
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
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Intervention(s)
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Genetic: Direct intravenous injection of ivlv-ADA lentiviral vector
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Primary Outcome(s)
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Overall survival up to a year
[Time Frame: 1 years]
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Secondary Outcome(s)
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2. Change of infection status
[Time Frame: 12 month]
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1. Success of immune reconstitution
[Time Frame: 12 month]
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Secondary ID(s)
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GIMI-IRB-18003
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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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