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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: 21 September 2015
Main ID:  NCT01279720
Date of registration: 18/01/2011
Prospective Registration: No
Primary sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
Public title: Gene Therapy ADA Deficiency
Scientific title: Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency
Date of first enrolment: October 2003
Target sample size: 8
Recruitment status: Completed
Study type:  Interventional
Study design:  Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 1/Phase 2
Countries of recruitment
United Kingdom
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patients who lack a human leukocyte antigen (HLA)-genotypically identical bone
marrow donor OR phenotypically matched family or unrelated donor AND who show
incomplete immune reconstitution on Polyethylene glycol-modified adenosine deaminase
(PEG-ADA) enzyme replacement therapy (defined by absolute CD4+ count <300 cell/mm3
and who remain on immunoglobulin replacement therapy)

2. Diagnosis of ADA-SCID (Severe combined immunodeficiency (SCID) due to adenosine
deaminase (ADA)confirmed by DNA sequencing OR by confirmed absence of <3% of ADA
enzymatic activity in peripheral blood or (for neonates) in umbilical cord blood
erythrocytes and/or leukocytes or in cultured fetal cells derived from either
chorionic villus biopsy or amniocentesis, prior to institution of PEG-ADA replacement

3. Parental/guardian/patient signed informed consent

Age minimum: N/A
Age maximum: 18 Years
Gender: Both
Health Condition(s) or Problem(s) studied
Adenosine Deaminase Deficiency
Biological: Intravenous infusion of transduced cells
Primary Outcome(s)
Immunological reconstitution [Time Frame: 5 years]
Secondary Outcome(s)
Incidence of adverse reactions [Time Frame: 5 years]
Molecular characterisation of gene transfer [Time Frame: 5 years]
Normalisation of nutritional status, growth, and development [Time Frame: 5 years]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available:
Date Posted:
Date Completed:
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