Status:
COMPLETED
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Lead Sponsor:
St. Jude Children's Research Hospital
Conditions:
Wiskott-Aldrich Syndrome
Eligibility:
MALE
Up to 18 years
Phase:
PHASE1
Brief Summary
Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic hematopoietic stem cell transplantation. A mismatched family member is an option when no human leukocyte antigen (HL...
Detailed Description
Secondary Objectives in this trial include the following: * To estimate the survival of study recipients at one year after infusion of the T- and B-lymphocyte depleted stem cell graft. * To assess if...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
May 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2009
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT00160355
Start Date
May 1 2005
End Date
February 1 2009
Last Update
April 26 2017
Active Locations (1)
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1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105