University of California, Los Angeles
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.
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 vector, as part of the UCL/A- ADA clinical development program. This will fulfil the requirements for the monitoring of delayed adverse events set by regulatory agencies for subjects treated with gene therapies. The study will have the following specific objectives: * To characterize the long-term safety of the gene therapy treatment; * To characterize the long-term clinical efficacy of the gene…
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…
ADA-SCID patients previously treated with 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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