What this study is
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.
What's being tested
- autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
Intervention · BIOLOGICAL
ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
Study details
- 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)
- Sponsor
- University of California, Los Angeles
- Conditions
- Adenosine Deaminase Deficiency · Severe Combined Immunodeficiency (SCID)
- Enrolling
- 70 people
- Starts
- 2019-09-26
- Est. completion
- 2040-08
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex ALLHealthy volunteers Not acceptedYou may qualify if
- 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 can't join if
- There are no exclusion criteria for participation in this observational LTFU study.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (2)
2 openMattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
1 site outside the US
UCL Great Ormond Street Institute of Child Health
London, United Kingdom
Study team
Sponsor
University of California, Los Angeles
Study ID OTL-101-6
Study director
Donald B. Kohn, M.D.
University of Califorina, Los Angeles
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.