19. Lysosomal storage disease Clinical trials / Disease details

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

15 trials found

diseases per page:

Showing 1 to 10 of 15 diseases

Filter by

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	NCT02800070 (ClinicalTrials.gov)	July 2016	15/4/2016	Autologous Stem Cell Transplantationof Cells Engineered to Express Alpha-Galactosidase A in Patients ...	Clinical Pilot Study of Autologous Stem Cell Transplantationof Cluster of Differentiation 34 Positiv ...	Fabry Disease	Biological: Lentivirus Alpha-gal A transduced stem cells	University Health Network, Toronto	Ozmosis Research Inc.	Active, not recruiting	18 Years	50 Years	Male	5	Phase 1	Canada
3	EUCTR2015-003031-35-GB (EUCTR)	20/11/2015	27/10/2015	Immune Tolerance Induction with Methotrexate in Hurler Syndrome	A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitig ...	Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH) MedDRA version: 18.1;Level: LLT;Classi ...	Product Name: Methotrexate INN or Proposed INN: Methotrexate	Central Manchester University Hospitals NHS Foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	4	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United Kingdom
4	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 Sever ...	Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome ...	Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparat ...	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	N/A	55 Years	All	100	Phase 2	United States
5	NCT01586455 (ClinicalTrials.gov)	April 2013	25/4/2012	Human Placental-Derived Stem Cell Transplantation	A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells C ...	Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Adrenoleukodystrophy;Niemann-Pick Disease;Metachrom ...	Drug: Human Placental Derived Stem Cell	New York Medical College	NULL	Completed	0 Years	55 Years	All	43	Phase 1	United States
6	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
7	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
8	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;T ...	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States
9	NCT01238315 (ClinicalTrials.gov)	November 2010	5/11/2010	Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis	A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Ne ...	Neuronal Ceroid Lipofuscinosis	Biological: HuCNS-SC	StemCells, Inc.	NULL	Withdrawn	6 Months	6 Years	Both	0	Phase 1	United States
10	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

First Previous1 2Next Last