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Clinical trial

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)

ID
Name
OTL-101-6
Description
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.
Trial arms
Trial start
2019-09-26
Estimated PCD
2035-08-01
Trial end
2035-08-01
Treatment
autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
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)
Size
70
Primary endpoint
Overall Survival
15 years post-treatment
Event-Free Survival
15 years post-treatment
Incidence of Adverse Events
15 years post-treatment
Eligibility criteria
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.
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'OTHER', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 70, 'type': 'ESTIMATED'}}
Updated at
2023-05-06

1 organization

1 product

2 indications

Organization
University of California, Los Angeles
Product
EFS-ADA LV
Indication
Adenosine Deaminase Deficiency
Indication
Severe Combined Immunodeficiency (SCID)