326. 大理石骨病
[臨床試験数:20,薬物数:52(DrugBank:14),標的遺伝子数:16,標的パスウェイ数:78

Searched query = "Osteopetrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
4 trials found
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1NCT02171104
(ClinicalTrials.gov)
July 10, 201420/6/2014MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis 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 DisordersBiological: 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 MinnesotaNULLRecruitingN/A55 YearsAll100Phase 2United States
2NCT01087398
(ClinicalTrials.gov)
September 200913/3/2010Hematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisHematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisOsteopetrosisDrug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)Tehran University of Medical SciencesNULLRecruitingN/A5 YearsBoth10Phase 2;Phase 3Iran, Islamic Republic of
3NCT00775931
(ClinicalTrials.gov)
August 200816/10/2008Allogeneic Transplantation For Severe OsteopetrosisAllogeneic Hematopoietic Stem Cell Transplantation For Severe OsteopetrosisSevere OsteopetrosisProcedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A45 YearsAll7Phase 2;Phase 3United States
4NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityNULLRecruitingN/A30 YearsBoth50Phase 2;Phase 3United States