Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

• 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 Malignant infantile osteopetrosis. 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: Busulfan; Cyclophosphamide; Cyclosporine; Cyclosporins; Fludarabine; Fludarabine phosphate; Methotrexate; Thymoglobulin

Criteria

Inclusion Criteria:

• Diagnosis of Osteopetrosis confirm by bone biopsy and radiographic imaging

• Age up to 5 year old

Exclusion Criteria:

• Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant

• Creatinine clearance ≤ 40ml/min/1.73m^2 or RTA

• Bilirubin ≥ 3mg/dL

• SGPT ≥ 500 U/L

• Current severe infection

• Evidence of CNS involvement

• Morbidity such as blindness or deafness