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

• Conditions: Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency (SCID)

• Registration Number: NCT04049084
• Lead Sponsor: University of California, Los Angeles

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-08-06
• Study First Submitted QC: 2019-08-06
• Study First Posted: 2019-08-07
• Study Start: 2019-09-26
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-03
• Last Update Posted: 2023-05-06
• Primary Completion: 2035-08
• Study Completion: 2035-08

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 70

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.

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Overall Survival	15 years post-treatment	Establishing efficacy through overall survival
Event-Free Survival	15 years post-treatment	Establishing efficacy through event-free survival
Incidence of Adverse Events	15 years post-treatment	Long-Term Safety incidence of AEs and SAEs

Trial Locations

Locations (2)

UCL Great Ormond Street Institute of Child Health; 🇬🇧 London, United Kingdom

Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center; 🇺🇸 Los Angeles, California, United States