Clinical trialNCT02064933CompletedObservational

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

SponsorNational Institute of Allergy and Infectious Diseases (NIAID)Enrollment305Wiskott-Aldrich Syndrome

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Alexandra H. Filipovich, MD
Children's Hospital Medical Center, Cincinnati
Study Chair
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David J. Rawlings, MD
Department of Pediatrics, University of Washington-Seattle Children's Hospital
Study Chair
clinicaltrials.gov ↗
Lauri M. Burroughs, MD
Fred Hutchinson Cancer Center
Study Chair
clinicaltrials.gov ↗
Luigi D. Notarangelo, MD
National Institute of Allergy and Infectious Diseases, NIH
Study Chair
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

43 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Canada

Alberta Children'S Hospital
Calgary, Alberta, Canada
Children'S & Women'S Health Centre of British Columbia
Vancouver, British Columbia, Canada
Cancer Care Manitoba, University of Manitoba
Winnipeg, Manitoba, Canada
the Hospital for Sick Children
Toronto, Ontario, Canada
Chu Sainte-Justine, Department of Pediatrics, University of Montreal
Montreal, Quebec, Canada

United States

Department of Pediatrics, University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Children'S Hospital
Phoenix, Arizona, United States
Cancer and Blood Disease Institute, Children'S Hospital Los Angeles, Keck School of Medicine, University of Southern California
Los Angeles, California, United States
Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles,
Los Angeles, California, United States
Lucile Salter Packard Children'S Hospital at Stanford
Palo Alto, California, United States
University of California, San Francisco Benioff Children'S Hospital
San Francisco, California, United States
Children'S Hospital Denver, University of Colorado
Denver, Colorado, United States
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States
Children'S National Hospital-George Washington University School of Medicine and Health Sciences
Washington D.C., District of Columbia, United States
Blood and Marrow Transplant Program, Johns Hopkins All Children'S Hospital
St. Petersburg, Florida, United States
Aflac Cancer and Blood Disorders Center, Emory/children'S Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
Center for Cancer and Blood Disorders, Children'S Hospital/louisiana State University
New Orleans, Louisiana, United States
Boston Children'S Hospital
Boston, Massachusetts, United States
Department of Pediatrics, C. S. Mott Children'S Hospital, University of Michigan
Ann Arbor, Michigan, United States
Division of Pediatric Blood and Marrow Transplantation, University of Minnesota
Minneapolis, Minnesota, United States
Mayo Clinic Children'S Center
Rochester, Minnesota, United States
Cardinal Glennon Children'S Hospital, Saint Louis University
St Louis, Missouri, United States
Saint Louis Children'S Hospital, Washington University
St Louis, Missouri, United States
Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center
Hackensack, New Jersey, United States
Department of Pediatrics, Memorial Sloan Kettering Cancer Center
New York, New York, United States
Department of Pediatrics, Golisano Children'S Hospital, University of Rochester
Rochester, New York, United States
Maria Fareri Children'S Hospital, New York Medical College
Valhalla, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children'S Hospital Medical Center, University of Cincinnati
Cincinnati, Ohio, United States
Rainbow Babies and Children'S Hospital
Cleveland, Ohio, United States
Nationwide Children'S Hospital
Columbus, Ohio, United States
Division of Pediatric Hematology/oncology, Oregon Health and Science University
Portland, Oregon, United States
Children'S Hospital of Philadelphia, University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh Children'S Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
St. Jude Children'S Research Hospital
Memphis, Tennessee, United States
Pediatrics, University of Texas Southwestern Medical Center
Dallas, Texas, United States
Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children'S Hospital
Houston, Texas, United States
Texas Transplant Institute, Methodist Children'S Hospital
San Antonio, Texas, United States
Primary Children'S Hospital, University of Utah
Salt Lake City, Utah, United States

3 more sites on the registry record.ClinicalTrials.gov ↗

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 2 Feb 2014
Primary completion: 1 May 2019
Completion: 1 May 2019
Last update posted: 10 Aug 2020
Registered enrollment: 305
Registry id: NCT02064933

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.