Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Phase 2

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

• Registration Number: NCT01087398
• Lead Sponsor: Tehran University of Medical Sciences

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 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-09
• Study First Submitted: 2010-03-13
• Study First Submitted QC: 2010-03-15
• Study First Posted: 2010-03-16
• Status Verified: 2012-05
• Last Update Submitted: 2012-05-31
• Last Update Posted: 2012-06-04
• Primary Completion: 2012-11
• Study Completion: 2012-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Intervention	Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)	-
Intervention	Stem Cell Transplantation	-
Intervention	Cyclosporin, Methotrexate (GVHD prophylaxis)	-

Primary Outcome Measures

Name	Time	Method
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT	1 year

Secondary Outcome Measures

Name	Time	Method
One year overall survival after allogeneic HSCT	1 year
One year Progressive Free Survival after allogeneic HSCT	1 year
Transplantation Related Mortality (TRM) after allogeneic HSCT	1 year
Acute and chronic GVHD rate after allogeneic HSCT	1 year

Trial Locations

Locations (1)

Hematology-Oncology & SCT Research Center; 🇮🇷 Tehran, Iran, Islamic Republic of