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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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15 May 2023 |
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Main ID: |
NCT04049084 |
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Date of registration:
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06/08/2019 |
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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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An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
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Scientific title:
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An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) |
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Date of first enrolment:
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September 26, 2019 |
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Target sample size:
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70 |
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Recruitment status: |
Enrolling by invitation |
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URL:
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https://clinicaltrials.gov/show/NCT04049084 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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United Kingdom
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United States
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Contacts
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Name:
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Orchard Clinical Trials |
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Address:
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Telephone:
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Email:
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Affiliation:
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Orchard Therapeutics |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
A patient is eligible for enrollment in the study if all of the following criteria are met:
1. the patient has been treated with an autologous ex vivo gene therapy product based on
the EFS-ADA LV, as part of the OTL-101 clinical development program;
2. the patient displays persistent detectable gene marking, as determined by the
Investigator;
3. the patient or, if applicable, the patient's parent(s)/legal guardian(s), are able and
willing to provide informed consent.
Exclusion Criteria:
- There are no exclusion criteria for participation in this observational LTFU study.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Severe Combined Immunodeficiency (SCID)
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Adenosine Deaminase Deficiency
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Intervention(s)
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Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
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Primary Outcome(s)
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Incidence of Adverse Events
[Time Frame: 15 years post-treatment]
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Overall Survival
[Time Frame: 15 years post-treatment]
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Event-Free Survival
[Time Frame: 15 years post-treatment]
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Secondary ID(s)
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OTL-101-6
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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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