An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Enrolling by invitation

Conditions studied: Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)

In brief

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.

Key facts

Study ID

NCT04049084

Run by

University of California, Los Angeles

People needed

70

Starts

2019-09-26

Expected to finish

2040-08-01

Last updated by the study team

2025-12-18

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• There are no exclusion criteria for participation in this observational LTFU study.

Where it is running

• Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center — Los Angeles, California, United States
• UCL Great Ormond Street Institute of Child Health — London, United Kingdom

Full record on ClinicalTrials.gov

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