Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

• Status: Unknown status
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

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 stem cell transplant for Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Tehran University of Medical Sciences
• Treatments: Antilymphocyte Serum; Thymoglobulin

Criteria

Inclusion Criteria:

• Diagnosis of Mucopolysaccharidosis type 1, 6 and 7 confirm by leukocyte enzyme assay

• Age up to 8 year old

• Have suitable donor

Exclusion Criteria:

• 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