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Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Primary Purpose

Osteopetrosis

Status
Unknown status
Phase
Phase 2
Locations
Iran, Islamic Republic of
Study Type
Interventional
Intervention
Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
Stem Cell Transplantation
Cyclosporin, Methotrexate (GVHD prophylaxis)
Sponsored by
Tehran University of Medical Sciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteopetrosis focused on measuring Osteopetrosis, HSCT

Eligibility Criteria

undefined - 5 Years (Child)All SexesDoes not accept 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

Sites / Locations

  • Hematology-Oncology & SCT Research CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Intervention

Arm Description

Outcomes

Primary Outcome Measures

Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT

Secondary Outcome Measures

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

Full Information

First Posted
March 13, 2010
Last Updated
May 31, 2012
Sponsor
Tehran University of Medical Sciences
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1. Study Identification

Unique Protocol Identification Number
NCT01087398
Brief Title
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
Official Title
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
Study Type
Interventional

2. Study Status

Record Verification Date
May 2012
Overall Recruitment Status
Unknown status
Study Start Date
September 2009 (undefined)
Primary Completion Date
November 2012 (Anticipated)
Study Completion Date
December 2012 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Tehran University of Medical Sciences

4. Oversight

Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteopetrosis
Keywords
Osteopetrosis, HSCT

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Intervention
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
Other Intervention Name(s)
Systemic chemotherapy
Intervention Description
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
Intervention Type
Procedure
Intervention Name(s)
Stem Cell Transplantation
Other Intervention Name(s)
HSCT
Intervention Description
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
Intervention Type
Drug
Intervention Name(s)
Cyclosporin, Methotrexate (GVHD prophylaxis)
Other Intervention Name(s)
Graft-versus-host disease (GVHD) prophylaxis
Intervention Description
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)
Primary Outcome Measure Information:
Title
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
Time Frame
1 year
Secondary Outcome Measure Information:
Title
One year overall survival after allogeneic HSCT
Time Frame
1 year
Title
One year Progressive Free Survival after allogeneic HSCT
Time Frame
1 year
Title
Transplantation Related Mortality (TRM) after allogeneic HSCT
Time Frame
1 year
Title
Acute and chronic GVHD rate after allogeneic HSCT
Time Frame
1 year

10. Eligibility

Sex
All
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Amir Ali Hamidieh, MD
Phone
84902645
Ext
+98-21
Email
aahamidieh@sina.tums.ac.ir
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amir Ali Hamidieh, MD
Organizational Affiliation
Hematology-Oncology and SCT Research Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hematology-Oncology & SCT Research Center
City
Tehran
ZIP/Postal Code
14114
Country
Iran, Islamic Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Amir Ali Hamidieh, MD
Phone
84902645
Ext
+98-21
Email
aahamidieh@sina.tums.ac.ir
First Name & Middle Initial & Last Name & Degree
Ardeshir Ghavamzadeh, MD
First Name & Middle Initial & Last Name & Degree
Mahdi Jalili, MD

12. IPD Sharing Statement

Learn more about this trial

Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

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