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

Plain-language summary

This observational study follows, over the long term, patients who were previously treated with an autologous ex vivo gene therapy (EFS-ADA LV carrying the human adenosine deaminase (ADA) gene) for ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency). It can enroll patients who received this gene therapy as part of the OTL-101 program and show persistent detectable gene marking. It is a study without an assigned phase and enrolls by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Enrolling by invitation

Sponsor

University of California, Los Angeles

Intervention

autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

Start date

9/26/2019

Estimated completion

8/1/2040

Countries

United States, United Kingdom

Related genes

Locations

• Los Angeles, United States
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.