234. ペルオキシソーム病(副腎白質ジストロフィーを除く。) Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details


臨床試験数 : 41 薬物数 : 37 - (DrugBank : 12) / 標的遺伝子数 : 12 - 標的パスウェイ数 : 43

  
1 trial found
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1NCT01626092
(ClinicalTrials.gov)
July 11, 201220/6/2012Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersTreatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)Lysosomal Storage Disease;Peroxisomal DisorderDrug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A55 YearsAll3N/AUnited States