An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Launched by UNIVERSITY OF CALIFORNIA, LOS ANGELES · Aug 6, 2019
Trial Information
Current as of August 22, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
No description provided
Gender
ALL
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.
About University Of California, Los Angeles
The University of California, Los Angeles (UCLA) is a prestigious academic institution renowned for its commitment to research and innovation in the biomedical field. As a clinical trial sponsor, UCLA leverages its cutting-edge facilities, expert faculty, and collaborative environment to advance medical knowledge and improve patient care. The university is dedicated to conducting rigorous clinical research that adheres to the highest ethical standards, aiming to translate scientific discoveries into effective therapies and interventions. Through its diverse array of clinical trials, UCLA seeks to address critical health challenges while fostering the development of future healthcare leaders.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Los Angeles, California, United States
London, , United Kingdom
Patients applied
Trial Officials
Orchard Clinical Trials
Study Director
Orchard Therapeutics
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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