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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

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : September 2009
Estimated Primary Completion Date : November 2012
Estimated Study Completion Date : December 2012

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Arms and Interventions
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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 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


Outcome Measures
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Primary Outcome Measures :
  1. Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. One year overall survival after allogeneic HSCT [ Time Frame: 1 year ]
  2. One year Progressive Free Survival after allogeneic HSCT [ Time Frame: 1 year ]
  3. Transplantation Related Mortality (TRM) after allogeneic HSCT [ Time Frame: 1 year ]
  4. Acute and chronic GVHD rate after allogeneic HSCT [ Time Frame: 1 year ]

Eligibility Criteria
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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01087398


Contacts
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Contact: Amir Ali Hamidieh, MD 84902645 ext +98-21 aahamidieh@sina.tums.ac.ir

Locations
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Iran, Islamic Republic of
Hematology-Oncology & SCT Research Center Recruiting
Tehran, Iran, Islamic Republic of, 14114
Contact: Amir Ali Hamidieh, MD    84902645 ext +98-21    aahamidieh@sina.tums.ac.ir   
Sub-Investigator: Ardeshir Ghavamzadeh, MD         
Sub-Investigator: Mahdi Jalili, MD         
Sponsors and Collaborators
Tehran University of Medical Sciences
Investigators
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Principal Investigator: Amir Ali Hamidieh, MD Hematology-Oncology and SCT Research Center
More Information
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Responsible Party: Tehran University of Medical Sciences
ClinicalTrials.gov Identifier: NCT01087398    
Other Study ID Numbers: HORCSCT-0905
First Posted: March 16, 2010    Key Record Dates
Last Update Posted: June 4, 2012
Last Verified: May 2012
Keywords provided by Tehran University of Medical Sciences:
Osteopetrosis
HSCT
Additional relevant MeSH terms:
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Osteopetrosis
Osteosclerosis
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Cyclosporine
Cyclophosphamide
Busulfan
Methotrexate
Fludarabine
Cyclosporins
Thymoglobulin
Immunosuppressive Agents
Immunologic Factors
 Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Antimetabolites, Antineoplastic
Antimetabolites
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists