326. 大理石骨病 Osteopetrosis Clinical trials / Disease details
臨床試験数 : 18 / 薬物数 : 43 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 82
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 2 | NCT01087398 (ClinicalTrials.gov) | September 2009 | 13/3/2010 | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Osteopetrosis | Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) | Tehran University of Medical Sciences | NULL | Recruiting | N/A | 5 Years | Both | 10 | Phase 2/Phase 3 | Iran, Islamic Republic of |
| 3 | NCT00775931 (ClinicalTrials.gov) | August 2008 | 16/10/2008 | Allogeneic Transplantation For Severe Osteopetrosis | Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis | Severe Osteopetrosis | Procedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantation | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 45 Years | All | 7 | Phase 2/Phase 3 | United States |
| 4 | NCT01019876 (ClinicalTrials.gov) | June 2002 | 23/11/2009 | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases | Bone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined Immunodeficiency | Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30 | Columbia University | NULL | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States |