Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intervention | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (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 infantile Osteopetrosis who receive allogeneic HSCT | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| One year overall survival after allogeneic HSCT | 1 year | |
| One year Progressive Free Survival after allogeneic HSCT | 1 year | |
| Transplantation Related Mortality (TRM) after allogeneic HSCT |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Amir Ali Hamidieh, MD | Contact | 84902645 | +98-21 | aahamidieh@sina.tums.ac.ir |
| Name | Affiliation | Role |
|---|---|---|
| Amir Ali Hamidieh, MD | Hematology-Oncology and SCT Research Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hematology-Oncology & SCT Research Center | Recruiting | Tehran | Tehran Province | 14114 | Iran |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
|
| Stem Cell Transplantation | Procedure | Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:
|
|
|
| Cyclosporin, Methotrexate (GVHD prophylaxis) | Drug |
|
|
|
| 1 year |
| Acute and chronic GVHD rate after allogeneic HSCT | 1 year |
| ID | Term |
|---|---|
| D010022 | Osteopetrosis |
| ID | Term |
|---|---|
| D010026 | Osteosclerosis |
| D010009 | Osteochondrodysplasias |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D002066 | Busulfan |
| D003520 | Cyclophosphamide |
| C512542 | thymoglobulin |
| C024352 | fludarabine |
| D019172 | Transplantation Conditioning |
| D020360 | Neoadjuvant Therapy |
| D033581 | Stem Cell Transplantation |
| D016572 | Cyclosporine |
| D008727 | Methotrexate |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D007165 | Immunosuppression Therapy |
| D007167 | Immunotherapy |
| D056747 | Immunomodulation |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D007158 | Immunologic Techniques |
| D008919 | Investigative Techniques |
| D003131 | Combined Modality Therapy |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000630 | Aminopterin |
| D011622 | Pterins |
| D011621 | Pteridines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
Not provided
Not provided