MedTrace Logo

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

NCT02064933 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 305

Last updated 2020-08-10

No results posted yet for this study

Summary

Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with WAS to be very susceptible to infections. Depending on the specific type of primary immune deficiency diseases, there are effective treatments, including antibiotics, cellular therapy and gene therapy, but studies of large numbers of patients are needed to determine the full range of causes, natural history, or the best methods of treatment for long term success.

This multicenter study combines retrospective, prospective and cross-sectional analyses of the transplant experiences for patients with WAS who have already received HCT since 1990, or who will undergo Hematopoietic cell transplant (HCT) during the study period. The retrospective and prospective portions of the study will address the impact of a number of pre and post-transplant factors on post-transplant disease correction and ultimate benefit from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years post-HCT in consenting surviving patients.

Conditions

  • Wiskott-Aldrich Syndrome

Sponsors & Collaborators

  • Primary Immune Deficiency Treatment Consortium (PIDTC)

    collaborator OTHER

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Lauri M. Burroughs, MD · Fred Hutchinson Cancer Center

  • David J. Rawlings, MD · Department of Pediatrics, University of Washington-Seattle Children's Hospital

  • Luigi D. Notarangelo, MD · National Institute of Allergy and Infectious Diseases, NIH

  • Alexandra H. Filipovich, MD · Children's Hospital Medical Center, Cincinnati

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-02
Primary Completion
2019-05-01
Completion
2019-05-01

Countries

  • United States
  • Canada

Study Locations

More Related Trials

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 NCT02064933 on ClinicalTrials.gov