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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT01087398 |
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Date of registration:
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13/03/2010 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
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Scientific title:
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Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis |
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Date of first enrolment:
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September 2009 |
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Target sample size:
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10 |
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Recruitment status: |
Recruiting |
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URL:
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http://clinicaltrials.gov/show/NCT01087398 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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Iran, Islamic Republic of
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Contacts
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Name:
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Amir Ali Hamidieh, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Hematology-Oncology and SCT Research Center |
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Name:
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Amir Ali Hamidieh, MD |
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Address:
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Telephone:
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84902645 |
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Email:
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aahamidieh@sina.tums.ac.ir |
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Affiliation:
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Name:
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Amir Ali Hamidieh, MD |
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Address:
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Telephone:
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84902645 |
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Email:
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aahamidieh@sina.tums.ac.ir |
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Affiliation:
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Key inclusion & exclusion criteria
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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
Age minimum:
N/A
Age maximum:
5 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Osteopetrosis
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Intervention(s)
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Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)
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Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
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Procedure: Stem Cell Transplantation
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Primary Outcome(s)
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Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
[Time Frame: 1 year]
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Secondary Outcome(s)
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Transplantation Related Mortality (TRM) after allogeneic HSCT
[Time Frame: 1 year]
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Acute and chronic GVHD rate after allogeneic HSCT
[Time Frame: 1 year]
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One year Progressive Free Survival after allogeneic HSCT
[Time Frame: 1 year]
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One year overall survival after allogeneic HSCT
[Time Frame: 1 year]
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Secondary ID(s)
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HORCSCT-0905
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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