19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details

臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

3 trials found

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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	NCT01626092 (ClinicalTrials.gov)	July 11, 2012	20/6/2012	Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorde ...	Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoi ...	Lysosomal Storage Disease;Peroxisomal Disorder	Drug: Campath -1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boo ...	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	55 Years	All	3	N/A	United States
2	NCT01399047 (ClinicalTrials.gov)	July 2011	5/7/2011	Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis	Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolatein Chi ...	Juvenile Neuronal Ceroid Lipofuscinosis	Drug: Mycophenolate mofetil;Drug: Liquid Placebo	University of Rochester	Batten Disease Support and Research Assocation (BDSRA)	Completed	6 Years	25 Years	All	19	Phase 2	United States
3	NCT01043640 (ClinicalTrials.gov)	December 2009	5/1/2010	Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders	Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Man ...	Drug: Campath -1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantati ...	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	21 Years	All	46	Phase 2	United States

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