A Study Analyzing Patients Treated for Wiskott-Aldrich Syndrome Since 1990

Protocol
14-066
Full Title
Analysis of Patients Treated for Wiscott-Aldrich Syndrome Since January 1, 1990
Disease Status
Newly Diagnosed & Relapsed/Refractory
Purpose

Wiskott-Aldrich syndrome (WAS) is a rare primary immune deficiency (PID). Because PIDs are so rare, medical centers have banded together into the Rare Disease Clinical Research Network-Primary Immune Deficiency Treatment Consortium. Memorial Sloan Kettering Cancer Center is part of this research group, which is conducting this study.

In one part of this study, researchers want to learn more about WAS by analyzing patients who received treatment for WAS (with either a bone marrow transplant or gene therapy) at least two years before enrolling in the study. The other part of this study will enroll patients who are newly diagnosed. These patients will be followed as they continue onward to get their treatment. Researchers will collect medical information and samples at routine medical visits to Memorial Sloan Kettering. Patients and their parents will also be asked to complete questionnaires about their quality of life.

The results of this study will help doctors better understand WAS and how to diagnose and treat it most effectively.

Eligibility

Participants in this study include:

  • Patients who were treated for WAS since 1990 with a bone marrow transplant or gene therapy completed at least two years before entering the study.
  • Patients who are newly diagnosed with WAS and will be receiving medical care and treatment.

For more information about this study, please contact Dr. Susan Prockop at 212-639-6715.

Disease(s)
Hematology: Hematologic Disorders
Locations