Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

This study is currently recruiting participants.
Verified May 2012 by Tehran University of Medical Sciences
Sponsor:
Information provided by (Responsible Party):
Tehran University of Medical Sciences
ClinicalTrials.gov Identifier:
NCT01087398
First received: March 13, 2010
Last updated: May 31, 2012
Last verified: May 2012
  Purpose

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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Resource links provided by NLM:


Further study details as provided by Tehran University of Medical Sciences:

Primary Outcome Measures:
  • Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • One year overall survival after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • One year Progressive Free Survival after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Transplantation Related Mortality (TRM) after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Acute and chronic GVHD rate after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: September 2009
Estimated Study Completion Date: December 2012
Estimated Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   up to 5 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01087398

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

Locations
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
Principal Investigator: Amir Ali Hamidieh, MD Hematology-Oncology and SCT Research Center
  More Information

Additional Information:
No publications provided

Responsible Party: Tehran University of Medical Sciences
ClinicalTrials.gov Identifier: NCT01087398     History of Changes
Other Study ID Numbers: HORCSCT-0905
Study First Received: March 13, 2010
Last Updated: May 31, 2012
Health Authority: Iran: Ministry of Health

Keywords provided by Tehran University of Medical Sciences:
Osteopetrosis
HSCT

Additional relevant MeSH terms:
Osteopetrosis
Osteosclerosis
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Busulfan
Cyclophosphamide
Cyclosporins
Cyclosporine
Methotrexate
Fludarabine monophosphate
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Therapeutic Uses
Myeloablative Agonists
Antirheumatic Agents
Enzyme Inhibitors
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Abortifacient Agents, Nonsteroidal
Abortifacient Agents

ClinicalTrials.gov processed this record on April 15, 2014