Status:
ENROLLING_BY_INVITATION
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Lead Sponsor:
University of California, Los Angeles
Collaborating Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust
Conditions:
Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency (SCID)
Eligibility:
All Genders
Brief Summary
This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-AD...
Detailed Description
The overall aim of this study is to record and evaluate long-term safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA ...
Eligibility Criteria
Inclusion
- A patient is eligible for enrollment in the study if all of the following criteria are met:
- 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;
- the patient displays persistent detectable gene marking, as determined by the Investigator;
- the patient or, if applicable, the patient's parent(s)/legal guardian(s), are able and willing to provide informed consent.
Exclusion
- There are no exclusion criteria for participation in this observational LTFU study.
Key Trial Info
Start Date :
September 26 2019
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 1 2040
Estimated Enrollment :
70 Patients enrolled
Trial Details
Trial ID
NCT04049084
Start Date
September 26 2019
End Date
August 1 2040
Last Update
December 18 2025
Active Locations (2)
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1
Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles, California, United States, 90095
2
UCL Great Ormond Street Institute of Child Health
London, United Kingdom, WC1N 1EH