Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Sponsor

Tehran University of Medical Sciences (Other)

Overall Status

Unknown status

CT.gov ID

NCT01087398

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Osteopetrosis	Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen) Procedure: Stem Cell Transplantation Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

10 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Study Start Date :

Sep 1, 2009

Anticipated Primary Completion Date :

Nov 1, 2012

Anticipated Study Completion Date :

Dec 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Intervention	Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen) For sibling full match: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg For haploidentical: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv Fludarabine 160 mg/m^2 Other Names: Systemic chemotherapy Procedure: Stem Cell Transplantation Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source: Sibling full match Other related full match Sibling or other related with 1 mismatch antigen Cord Blood Haploidentical Other Names: HSCT Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po 10 mg/m^2 iv day +1 then 6 mg/m^ iv day +3 and +6 (Not for UCBT) Other Names: Graft-versus-host disease (GVHD) prophylaxis

Arm

Intervention/Treatment

Experimental: Intervention

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

For sibling full match: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg For haploidentical: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv Fludarabine 160 mg/m^2

Other Names:

Systemic chemotherapy

Procedure: Stem Cell Transplantation

Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source: Sibling full match Other related full match Sibling or other related with 1 mismatch antigen Cord Blood Haploidentical

Other Names:

HSCT

Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)

Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po 10 mg/m^2 iv day +1 then 6 mg/m^ iv day +3 and +6 (Not for UCBT)

Other Names:

Graft-versus-host disease (GVHD) prophylaxis