Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
Public ClinicalTrials.gov record NCT02064933. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT02064933
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 305 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 2, 2014 (Actual)
- Primary completion
- May 1, 2019 (Actual)
- Completion
- May 1, 2019 (Actual)
- Last update posted
- Aug 10, 2020
2014 – 2019
United States locations
- U.S. sites
- 38
- U.S. states
- 25
- U.S. cities
- 36
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Department of Pediatrics, University of Alabama at Birmingham | Birmingham | Alabama | 35233 | — |
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California | Los Angeles | California | 90027 | — |
| Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, | Los Angeles | California | 90095-1752 | — |
| Lucile Salter Packard Children's Hospital at Stanford | Palo Alto | California | 94304 | — |
| University of California, San Francisco Benioff Children's Hospital | San Francisco | California | 94143-1278 | — |
| Children's Hospital Denver, University of Colorado | Denver | Colorado | 80220 | — |
| Nemours Alfred I. duPont Hospital for Children | Wilmington | Delaware | 19803 | — |
| Children's National Hospital-George Washington University School of Medicine and Health Sciences | Washington D.C. | District of Columbia | 20010-2970 | — |
| Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital | St. Petersburg | Florida | 33701 | — |
| Aflac Cancer and Blood Disorders Center, Emory/Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60614 | — |
| Center for Cancer and Blood Disorders, Children's Hospital/Louisiana State University | New Orleans | Louisiana | 70118 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Department of Pediatrics, C. S. Mott Children's Hospital, University of Michigan | Ann Arbor | Michigan | 48109 | — |
| Division of Pediatric Blood and Marrow Transplantation, University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Mayo Clinic Children's Center | Rochester | Minnesota | 55905 | — |
| Cardinal Glennon Children's Hospital, Saint Louis University | St Louis | Missouri | 63104 | — |
| Saint Louis Children's Hospital, Washington University | St Louis | Missouri | 63110 | — |
| Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Department of Pediatrics, Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | — |
| Department of Pediatrics, Golisano Children's Hospital, University of Rochester | Rochester | New York | 14642 | — |
| Maria Fareri Children's Hospital, New York Medical College | Valhalla | New York | 10595 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Cincinnati Children's Hospital Medical Center, University of Cincinnati | Cincinnati | Ohio | 45229-3039 | — |
| Rainbow Babies and Children's Hospital | Cleveland | Ohio | 44106 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Division of Pediatric Hematology/Oncology, Oregon Health and Science University | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia, University of Pennsylvania Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
| Pediatrics, University of Texas Southwestern Medical Center | Dallas | Texas | 75390-9263 | — |
| Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children's Hospital | Houston | Texas | 77030-2399 | — |
| Texas Transplant Institute, Methodist Children's Hospital | San Antonio | Texas | 78229 | — |
| Primary Children's Hospital, University of Utah | Salt Lake City | Utah | 84113 | — |
| Fred Hutchinson Cancer Research Center and University of Washington-Seattle Children's Hospital | Seattle | Washington | 98109 | — |
| American Family Children's Hospital, University of Wisconsin | Madison | Wisconsin | 53705-2275 | — |
| Children's Hospital of Wisconsin-Milwaukee | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02064933, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 10, 2020 · Synced Oct 26, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02064933 live on ClinicalTrials.gov.