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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 12 December 2020
Main ID:  NCT04049084
Date of registration: 06/08/2019
Prospective Registration: Yes
Primary sponsor: Orchard Therapeutics
Public title: An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Scientific 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)
Date of first enrolment: September 26, 2019
Target sample size: 70
Recruitment status: Enrolling by invitation
Study type:  Observational
Study design:   
Countries of recruitment
United Kingdom United States
Name:     Orchard Clinical Trials
Affiliation:  Orchard Therapeutics
Key inclusion & exclusion 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

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.

Age minimum: N/A
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency (SCID)
Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
Primary Outcome(s)
Event-Free Survival [Time Frame: 15 years post-treatment]
Incidence of Adverse Events [Time Frame: 15 years post-treatment]
Overall Survival [Time Frame: 15 years post-treatment]
Secondary Outcome(s)
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Great Ormond Street Hospital for Children NHS Foundation Trust
University of California, Los Angeles
Ethics review
Results available:
Date Posted:
Date Completed:
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