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Clinical Trial 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.


Clinical Trial Description

n/a


Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT01087398
Study type Interventional
Source Tehran University of Medical Sciences
Contact Amir Ali Hamidieh, MD
Phone 84902645
Email aahamidieh@sina.tums.ac.ir
Status Recruiting
Phase Phase 2/Phase 3
Start date September 2009
Completion date December 2012

See also
  Status Clinical Trial Phase
Terminated NCT00638820 - Reduced Intensity AlloTransplant For Osteopetrosis Phase 2
Completed NCT00004402 - Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis Phase 3
Recruiting NCT01019876 - Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases Phase 2/Phase 3
Terminated NCT00145587 - Stem Cell Transplantation for Children Affected With Osteopetrosis N/A
Completed NCT01199094 - Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5 N/A
Completed NCT00043329 - Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis Phase 4
Recruiting NCT03333200 - Longitudinal Study of Neurodegenerative Disorders
Completed NCT02666768 - ACTIMMUNE in Intermediate Osteopetrosis Phase 2
Terminated NCT00145886 - rhPTH Therapy for Low Turnover Bone Fragility Phase 1
Terminated NCT00968864 - T-cell Depleted Alternative Donor Transplantation Phase 2
Completed NCT00730314 - Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells Phase 1/Phase 2
Active, not recruiting NCT03301168 - Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant Phase 1/Phase 2
Terminated NCT02065869 - Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant Phase 1/Phase 2
No longer available NCT01200017 - Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant