ClinicalTrials.gov
ClinicalTrials.gov Menu

Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01087398
Recruitment Status : Unknown
Verified May 2012 by Tehran University of Medical Sciences.
Recruitment status was:  Recruiting
First Posted : March 16, 2010
Last Update Posted : June 4, 2012
Sponsor:
Information provided by (Responsible Party):
Tehran University of Medical Sciences

March 13, 2010
March 16, 2010
June 4, 2012
September 2009
November 2012   (Final data collection date for primary outcome measure)
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT [ Time Frame: 1 year ]
Same as current
Complete list of historical versions of study NCT01087398 on ClinicalTrials.gov Archive Site
  • One year overall survival after allogeneic HSCT [ Time Frame: 1 year ]
  • One year Progressive Free Survival after allogeneic HSCT [ Time Frame: 1 year ]
  • Transplantation Related Mortality (TRM) after allogeneic HSCT [ Time Frame: 1 year ]
  • Acute and chronic GVHD rate after allogeneic HSCT [ Time Frame: 1 year ]
Same as current
Not Provided
Not Provided
 
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
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.
Not Provided
Interventional
Phase 2
Phase 3
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Osteopetrosis
  • 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 Name: Systemic chemotherapy
  • Procedure: Stem Cell Transplantation

    Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:

    1. Sibling full match
    2. Other related full match
    3. Sibling or other related with 1 mismatch antigen
    4. Cord Blood
    5. Haploidentical
    Other Name: 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 Name: Graft-versus-host disease (GVHD) prophylaxis
Experimental: Intervention
Interventions:
  • Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
  • Procedure: Stem Cell Transplantation
  • Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Unknown status
10
Same as current
December 2012
November 2012   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
up to 5 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Iran, Islamic Republic of
 
 
NCT01087398
HORCSCT-0905
Yes
Not Provided
Not Provided
Tehran University of Medical Sciences
Tehran University of Medical Sciences
Not Provided
Principal Investigator: Amir Ali Hamidieh, MD Hematology-Oncology and SCT Research Center
Tehran University of Medical Sciences
May 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP