Search / Trial NCT00001255

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study

Launched by NATIONAL HUMAN GENOME RESEARCH INSTITUTE (NHGRI) · Nov 3, 1999

Trial Information

Current as of December 14, 2024

Completed

Keywords

Retroviral Vector Gene Therapy Peg Ada Severe Combined Immunodeficiency (Scid) Adenosine Deaminase Deficiency (Ada)

ClinConnect Summary

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.

Gender

ALL

Eligibility 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.

About National Human Genome Research Institute (Nhgri)

The National Human Genome Research Institute (NHGRI) is a prominent research organization within the National Institutes of Health (NIH) dedicated to advancing the field of genomics and its applications in health and medicine. NHGRI sponsors and conducts a wide range of clinical trials aimed at understanding the genetic basis of diseases, developing innovative genomic technologies, and translating genomic research into clinical practice. With a commitment to ethical research and collaboration, NHGRI plays a pivotal role in shaping the future of personalized medicine and improving patient outcomes through genomic discoveries.

Locations

Bethesda, Maryland, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Discussion 0

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