Trials / Conditions / Lysosomal Storage Diseases

Lysosomal Storage Diseases

20 registered clinical trials studyying Lysosomal Storage Diseases — 2 currently recruiting.

Status	Trial	Sponsor	Phase
Unknown	Nutritional Therapy in Late-onset Pompe Disease NCT06130228	McMaster University	Phase 2
Active Not Recruiting	OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) NCT04283227	Orchard Therapeutics	Phase 3
Active Not Recruiting	Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clin NCT04943991	Wuerzburg University Hospital	N/A
Recruiting	A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry NCT04393701	University Hospital, Rouen	N/A
Active Not Recruiting	A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) NCT04093349	Spark Therapeutics, Inc.	Phase 1 / Phase 2
Withdrawn	Complement Activation in the Lysosomal Storage Disorders NCT04189601	Melbourne Health	—
Completed	A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 NCT04455230	Spur Therapeutics	Phase 1 / Phase 2
Completed	Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders NCT04399694	Duke University	—
Completed	Stem Cell Gene Therapy for Cystinosis NCT03897361	University of California, San Diego	Phase 1 / Phase 2
Terminated	A Fabry Disease Gene Therapy Study NCT04040049	Spur Therapeutics	Phase 1 / Phase 2
Completed	Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pomp NCT03893240	Spark Therapeutics, Inc.	N/A
Terminated	A Natural History Study of Aspartylglucosaminuria NCT03853876	Neurogene Inc.	—
Completed	Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease NCT02416661	CENTOGENE GmbH Rostock	—
Unknown	Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders NCT03812055	Lysosomal and Rare Disorders Research and Treatment Center, Inc.	—
Completed	Diet and Exercise in Pompe Disease NCT02363153	University of Florida	N/A
Unknown	Screening of Lysosomal Storage Disorders Diseases in Minority Groups NCT03812042	Lysosomal and Rare Disorders Research and Treatment Center, Inc.	—
Terminated	Natural History Study of Children With Metachromatic Leukodystrophy NCT01963650	Shire	—
Recruiting	Longitudinal Study of Neurodegenerative Disorders NCT03333200	University of Pittsburgh	—
Withdrawn	Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases NCT01003912	Joanne Kurtzberg, MD	Phase 1
Terminated	Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I NCT00215527	Patricia I. Dickson, M.D.	Phase 1