This study is accepting new patients by invitation only

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

a study on ADA Deficiency Severe Combined Immunodeficiency Immunodeficiency

Summary

Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Donald B. Kohn, M.D. (ucla)

Description

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.

Official Title

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)

Keywords

Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID), OTL-101, Severe Combined Immunodeficiency, Immunologic Deficiency Syndromes, Adenosine, autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

Eligibility

You can join if…

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.

You CAN'T join if...

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

Locations

  • Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
    Los Angeles California 90095 United States
  • UCL Great Ormond Street Institute of Child Health
    London WC1N 1EH United Kingdom

Lead Scientist at University of California Health

  • Donald B. Kohn, M.D. (ucla)
    Professor, MIMG, Medicine. Authored (or co-authored) 254 research publications

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
University of California, Los Angeles
ID
NCT04049084
Study Type
Observational
Participants
Expecting 70 study participants
Last Updated