19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

9 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	NCT05739643 (ClinicalTrials.gov)	February 3, 2023	13/2/2023	Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated in the Past Wit ...	A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previ ...	Krabbe Disease	Biological: FBX-101	Forge Biologics, Inc	NULL	Recruiting	N/A	N/A	All	12	Phase 1/Phase 2	United States
2	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
3	NCT01572636 (ClinicalTrials.gov)	March 28, 2012	4/4/2012	Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hu ...	MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell ...	Mucopolysaccharidosis Type IH;MPS I;Hurler Syndrome	Drug: Laronidase	Masonic Cancer Center, University of Minnesota	NULL	Terminated	N/A	N/A	All	20		United States
4	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
5	NCT01303146 (ClinicalTrials.gov)	October 2008	23/2/2011	Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell ...	and Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients Wit ...	Metachromatic Leukodystrophy	Drug: rhARSA	Assistance Publique - Hôpitaux de Paris	European Leukodystrophy Association;Zymenex A/S;Shire	Completed	6 Months	N/A	Both	1	Phase 2	France
6	NCT00668564 (ClinicalTrials.gov)	March 2008	25/4/2008	Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism	Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplanta ...	Hurler's Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucos ...	Procedure: Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: Campath -1H;Drug: Busulfan	Masonic Cancer Center, University of Minnesota	NULL	Terminated	N/A	21 Years	All	18	Phase 2	United States
7	NCT00383448 (ClinicalTrials.gov)	September 2006	29/9/2006	HSCT for High Risk Inherited Inborn Errors	Treatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoi ...	Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Tay Sachs Disease;Sand ...	Drug: Clofarabine;Procedure: Total body Irradiation;Drug: Melphalan;Biological: Hematopoietic Stem C ...	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	70 Years	All	38	Phase 2	United States
8	NCT00176891 (ClinicalTrials.gov)	March 2004	12/9/2005	Stem Cell Transplant w/Laronidase for Hurler	Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoi ...	Mucopolysaccharidosis I;Hurler Syndrome	Procedure: Stem Cell Transplant;Drug: Laronidase ERT	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	7 Years	All	25	Phase 2	United States
9	NCT00176917 (ClinicalTrials.gov)	May 1999	12/9/2005	Stem Cell Transplantation for Hurler	Hematopoietic Stem Cell Transplantationfor Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Al ...	Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Mannosidosis;Mucolipidosis Type II (I-cell Disease) ...	Procedure: Stem Cell Transplant;Drug: Busulfan , Cyclophosphamide, ATG	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	N/A	All	41	Phase 2	United States

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