Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome
About this trial
This is an interventional treatment trial for Wiskott-Aldrich Syndrome focused on measuring Wiskott-Aldrich Syndrome, Immunodeficiency, Haploidentical transplantation
Eligibility Criteria
Inclusion Criteria: Genotypical diagnosis of Wiskott-Aldrich Syndrome. Less than 18 years of age at time of transplant. Must meet two of the eight following clinical criteria: Eczema that is refractory to standard therapy. Thrombocytopenia as defined by a platelet count < 50,000/mm3. Significant risk for or presence of opportunistic infection. Autoimmune disease. Malignancy or pre-malignant condition. Family history as defined as a family member with WAS who died before 10 years of age. Does not have a suitable, available 6/6 HLA-matched sibling donor available for donation. Does not have a suitable, available 10/10 HLA-allele matched unrelated donor identified through the National Marrow Donor Program (NMDP). Exclusion Criteria: If any of the following clinical indicators are met within 45 days prior to transplant, the research participant will not be eligible for the study: Symptomatic cardiac disease or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction < 30%). Creatinine clearance or Tc 99 less than or equal 40ml/min/1.73 m2. SGPT greater than or equal 500 U/L. Karnofsky or Lansky Performance Score of < 50. Pulmonary function tests: FVC < 50% of predicted value if age appropriate to perform the testing adequately or an O2 saturation less than or equal to 92% on room air at rest.
Sites / Locations
- St. Jude Children's Research Hospital
Arms of the Study
Arm 1
Other
1