Completed

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

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What is being collected

Drug Data

+ drug Data

+ drug Data

Drug
Who is being recruted

Primary Immunodeficiency Diseases+7

+ Immunologic Deficiency Syndromes

+ Immune System Diseases

See all eligibility criteria
How is the trial designed

Observational
Study Start: September 1990
See protocol details

Summary

Principal SponsorNational Human Genome Research Institute (NHGRI)
Last updated: March 4, 2008
Sourced from a government-validated database.Claim as a partner

Study start date: September 1, 1990

Actual date on which the first participant was enrolled.

The primary purpose of this study is to continue to provide clinical follow-up for ADA-deficient patients treated with gene therapy under the original protocol 90-HG-0195 (IND 3624) and its amendments (IND 4647 and IND 5056). The objectives are the long-term monitoring of the beneficial effects of gene therapy and continued surveillance of potential adverse effects associated with the gene transfer procedures. No new subjects will be enrolled in this protocol.

NCT00001255
Principal SponsorNational Human Genome Research Institute (NHGRI)
Last updated: March 4, 2008
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

10 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Primary Immunodeficiency DiseasesImmunologic Deficiency SyndromesImmune System DiseasesInfant, Newborn, DiseasesMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNutritional and Metabolic DiseasesGenetic Diseases, InbornDNA Repair-Deficiency DisordersSevere Combined Immunodeficiency

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 procedures.

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

National Human Genome Research Institute (NHGRI)

Bethesda, United StatesOpen National Human Genome Research Institute (NHGRI) in Google Maps
CompletedOne Study Center