Status:
UNKNOWN
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Lead Sponsor:
Tehran University of Medical Sciences
Conditions:
Mucopolysaccharidosis
Eligibility:
All Genders
Up to 8 years
Phase:
PHASE2
PHASE3
Brief Summary
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic ste...
Eligibility Criteria
Inclusion
- Diagnosis of Mucopolysaccharidosis type 1, 6 and 7 confirm by leukocyte enzyme assay
- Age up to 8 year old
- Have suitable donor
Exclusion
- Creatinine clearance ≤ 40ml/min/1.73m\^2
- Bilirubin ≥ 3mg/dL
- SGPT ≥ 500 U/L
- Current severe infection
- Evidence of CNS involvement
- Morbidity such as blindness or deafness
Key Trial Info
Start Date :
September 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2012
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT01238328
Start Date
September 1 2009
End Date
December 1 2012
Last Update
June 4 2012
Active Locations (1)
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1
Hematology-Oncology & SCT Research Center
Teharn, Tehran Province, Iran, 14114