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Natural History Study of SCID Disorders

NCT01186913 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 690

Last updated 2020-11-10

No results posted yet for this study

Summary

This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study.

Children will be divided into three strata:

* Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function
* Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and
* Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy.

Each Group/Cohort Stratum will be analyzed separately.

Conditions

  • Severe Combined Immunodeficiency (SCID)
  • Leaky SCID
  • Omenn Syndrome
  • Reticular Dysgenesis
  • ADA SCID
  • XSCID

Sponsors & Collaborators

  • Primary Immune Deficiency Treatment Consortium (PIDTC)

    collaborator OTHER

  • Office of Rare Diseases (ORD)

    collaborator NIH

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Christopher C. Dvorak, MD · UCSF Children's Hospital

  • Morton J. Cowan, MD · UCSF Children's Hospital

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-09-02
Primary Completion
2028-09-30
Completion
2028-09-30

Countries

  • United States
  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01186913 on ClinicalTrials.gov