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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: 19 February 2015
Main ID:  NCT00001255
Date of registration: 03/11/1999
Prospective Registration: No
Primary sponsor: National Human Genome Research Institute (NHGRI)
Public title: Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
Scientific title: Treatment of Severe Combined Immunodeficiency Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency With Autologous Lymphocytes of CD34+ Cells Transduced With a Human ADA Gene: A Natural History Study
Date of first enrolment: September 1990
Target sample size: 10
Recruitment status: Completed
Study type:  Observational
Study design:  N/A  
Phase:  N/A
Countries of recruitment
United States
Key inclusion & exclusion criteria

New patients will not be treated under protocol 90-HG-0195 as new and improved vectors and
technologies have become available in the recent years.

New patients with ADA deficiency, however, may be enrolled in protocol 90-HG-0195 for
clinical evaluation of their immune system and pre-treatment testing of transduction

Age minimum: N/A
Age maximum: N/A
Gender: Both
Health Condition(s) or Problem(s) studied
Severe Combined Immunodeficiency
Drug: ADA Umbilical Cord Blood Cells
Drug: Transduced Lymphocytes
Primary Outcome(s)
Secondary Outcome(s)
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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