Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Tehran University of Medical Sciences

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Osteopetrosis

Treatments: Procedure: Stem Cell Transplantation; Drug: Cyclosporin, Methotrexate (GVHD prophylaxis); Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)

Study type: Interventional

Funder types: Other

Identifiers

NCT01087398

HORCSCT-0905

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: Under 5 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Intervention

Experimental group

Treatment:

Procedure: Stem Cell Transplantation

Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)

Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)