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

Completed

Conditions studied: Wiskott-Aldrich Syndrome

In brief

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.

Key facts

Study ID

NCT02064933

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

305

Starts

2014-02-02

Expected to finish

2019-05-01

Last updated by the study team

2020-08-10

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• WAS participants will be defined as males who have:
• thrombocytopenia (< 100K) AND EITHER molecular diagnosis of WAS OR reduced WASP expression; OR
• thrombocytopenia (< 100K) AND positive family history consistent with WAS diagnosis; OR
• chronic thrombocytopenia (< 100K for minimum of 3 months) AND low mean platelet volume (MPV below normal range for age) AND EITHER recurrent and/or severe infections requiring treatment and/or eczema OR lack of antibody response to polysaccharide antigens or low IgM.
• Longitudinal Analysis (Retrospective and Prospective)
• Stratum A. Participants with WAS who have or will Receive HCT
• Participants with WAS who have received an HCT since January 1, 1990
• Stratum B. Participants with WAS who have or will Receive Gene Transfer
• Participants in which the intention is to treat with gene transfer with autologous modified cells
• Cross-Sectional Analysis (Strata A and B) 1. Participants with WAS who are surviving and at least 2 years after the most recent HCT or gene therapy.

You may not qualify if…

• As this is a natural history study, for both the Longitudinal Analysis and the Cross-Sectional Analysis we will not exclude any patients due to race or age who fit the inclusion criteria.

Where it is running

• 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

Full record on ClinicalTrials.gov

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