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Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
NCT02064933 · View on ClinicalTrials.gov ↗
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 Studied
Study Locations (20)
California
- Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California — Los Angeles
- Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, — Los Angeles
- Lucile Salter Packard Children's Hospital at Stanford — Palo Alto
- University of California, San Francisco Benioff Children's Hospital — San Francisco
Minnesota
- Division of Pediatric Blood and Marrow Transplantation, University of Minnesota — Minneapolis
- Mayo Clinic Children's Center — Rochester
Missouri
- Cardinal Glennon Children's Hospital, Saint Louis University — St Louis
- Saint Louis Children's Hospital, Washington University — St Louis
Alabama
- Department of Pediatrics, University of Alabama at Birmingham — Birmingham
Arizona
- Phoenix Children's Hospital — Phoenix
Colorado
- Children's Hospital Denver, University of Colorado — Denver
Delaware
- Nemours Alfred I. duPont Hospital for Children — Wilmington
District of Columbia
- Children's National Hospital-George Washington University School of Medicine and Health Sciences — Washington D.C.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 305 participants |
| Start Date | 2014-02-02 |
| Est. Completion | 2019-05-01 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02064933
The ClinicalTrials.gov registry entry for NCT02064933 describes a study currently listed as completed. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 305 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Institute of Allergy and Infectious Diseases (NIAID), which has 1,295 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Wiskott-Aldrich Syndrome appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02064933 reports 20 study locations spanning 15 distinct geographic areas — top geographies include California, Minnesota, Missouri. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT02064933 about?
NCT02064933 is a clinical study titled "Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990". 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 deficienc...
What is the current status of trial NCT02064933?
This trial is currently completed. The enrollment target is 305 participants. The study started on 2014-02-02. Estimated completion is 2019-05-01.
What conditions does trial NCT02064933 study?
This clinical trial studies the following conditions: Wiskott-Aldrich Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT02064933?
This trial is sponsored by National Institute of Allergy and Infectious Diseases (NIAID), which has 1,295 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02064933 being conducted?
This trial has 20 study locations across Alabama, Arizona, California, Colorado, Delaware. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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