Trials / Conditions / Lysosomal Storage Disease

Lysosomal Storage Disease

10 registered clinical trials studyying Lysosomal Storage Disease.

Status	Trial	Sponsor	Phase
Withdrawn	Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) NCT01458613	CENTOGENE GmbH Rostock	—
Completed	A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) NCT03392987	Orchard Therapeutics	Phase 2
Completed	Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders NCT01626092	Masonic Cancer Center, University of Minnesota	N/A
Completed	Gene Therapy for Metachromatic Leukodystrophy (MLD) NCT01560182	Orchard Therapeutics	Phase 1 / Phase 2
Completed	A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I NCT00852358	Patricia I. Dickson, M.D.	N/A
Completed	Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Di NCT00744692	Duke University	Phase 1
Terminated	Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I NCT00786968	Patricia I. Dickson, M.D.	Phase 1
Completed	Study of Inborn Errors of Cholesterol Synthesis and Related Disorders NCT00046202	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	—
Completed	Magnetic Stimulation of the Human Nervous System NCT00001780	National Institute of Neurological Disorders and Stroke (NINDS)	—
Completed	The Classification and Cause of Leukodystrophies of Unknown Cause NCT00001671	National Institute of Neurological Disorders and Stroke (NINDS)	—