Trial record 1 of 1 for:
survival and GVHD after allogeneic HSCT for osteopetrosis
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
This study is currently recruiting participants.
Verified May 2012 by Tehran University of Medical Sciences
Sponsor:
Tehran University of Medical Sciences
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
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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:
Drug Information available for:
Cyclophosphamide
Busulfan
Methotrexate
Methotrexate sodium
Fludarabine
Cyclosporine
Fludarabine phosphate
U.S. FDA Resources
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:
For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood:
For haploidentical:
Other Name: Systemic chemotherapy
Procedure: Stem Cell Transplantation
Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:
Other Name: HSCT
Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)
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 May 16, 2013