Status:
COMPLETED
Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
Lead Sponsor:
The Korean Society of Pediatric Hematology Oncology
Conditions:
Wiskott-Aldrich Syndrome
Eligibility:
All Genders
1-25 years
Phase:
PHASE1
PHASE2
Brief Summary
Wiskott-Aldrich syndrome (WAS) is a rare X-linked congenital immune-deficiency syndrome and hematopoietic stem cell transplantation (HSCT) has become a curative modality. But the transplant with the c...
Detailed Description
Wiskott-Aldrich syndrome (WAS) is an rare X-linked congenital immune-deficiency syndrome characterized by the triad of recurrent infection, eczema and thrombocytopenia with small size of platelet (Puc...
Eligibility Criteria
Inclusion
- Diagnosis of Wiskott-Aldrich syndrome with gene analysis.
- Indicated for hematopoietic stem cell transplantation.
- Age: no limitation.
- Performance status: ECOG 0-2.
- Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases:
- Heart: a shortening fraction \> 30%, ejection fraction \> 45%.
- Liver: total bilirubin \< 2 × upper limit of normal; ALT \< 3 × upper limit of normal.
- Kidney: creatinine \<2 × normal or a creatinine clearance (GFR) \> 60 ml/min/1.73m2.
- Patients must lack any active viral infections or active fungal infection.
- Appropriate hematopoietic stem cell donor is available.
- Patients (or one of parents if patients age \< 19) should sign informed consent.
Exclusion
- Pregnant or nursing women.
- Malignant (except acute myeloid leukemia) or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
- Psychiatric disorder that would preclude compliance.
Key Trial Info
Start Date :
February 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2012
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT00885833
Start Date
February 1 2007
End Date
March 1 2012
Last Update
July 14 2014
Active Locations (1)
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1
Seoul National University Hospital
Seoul, Chongno-gu, South Korea, 110-744