An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
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ClinicalTrials.gov Identifier: NCT04049084
Recruitment Status :
Enrolling by invitation
First Posted : August 7, 2019
Last Update Posted : August 7, 2019
University of California, Los Angeles
Great Ormond Street Hospital for Children NHS Foundation Trust
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-ADA LV encoding for human adenosine deaminase (ADA) gene (EFS-ADA LV), as part of the OTL-101 clinical development program. No investigational medicinal product will be administered to these patients as part of the OTL-101-6 study.
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Layout table for eligibility information
Ages Eligible for Study:
Child, Adult, Older Adult
Sexes Eligible for Study:
Accepts Healthy Volunteers:
This observational LTFU study is open for enrollment to ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA lentiviral vector as part of the OTL-101 clinical development program. Patients treated in clinical trials as part of IND #15440, study OTL-101-5 (EudraCT No 2017-001275-23) or study 10-MI-29 (EudraCT No 2010-024253-36), as well as in Expanded Access or Compassionate Use Programs, will be eligible for enrollment.
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.
There are no exclusion criteria for participation in this observational LTFU study.