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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 Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Transplantation | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Conditioning regimen | Drug | For sibling full match:
For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood:
For haploidentical:
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT) | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT) | 1 year | |
| One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT) | 1 year |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Amir Ali Hamidieh, MD | Contact | 84902645 | +98 21 | aahamidieh@sina.tums.ac.ir |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hematology-Oncology & SCT Research Center | Recruiting | Teharn | Tehran Province | 14114 | Iran |
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| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| ID | Term |
|---|---|
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D019172 | Transplantation Conditioning |
| D003520 | Cyclophosphamide |
| D000961 | Antilymphocyte Serum |
| D033581 | Stem Cell Transplantation |
| ID | Term |
|---|---|
| D007165 | Immunosuppression Therapy |
| D007167 | Immunotherapy |
| D056747 | Immunomodulation |
| D001691 | Biological Therapy |
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|
|
| Graft-versus-host disease (GVHD) prophylaxis | Drug |
|
|
|
| Stem Cell Transplantation | Procedure | Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:
|
|
|
| Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT) | 1 year |
| Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT) | 1 year |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D013812 |
| Therapeutics |
| D007158 | Immunologic Techniques |
| D008919 | Investigative Techniques |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |