lysosomal storage diseases
Showing 1 - 16 of 16
Pompe Disease, Muscle Loss, Obesity Trial (Multi-ingredient supplement (PDT-MIS), Placebo (PLA))
Not yet recruiting
- Pompe Disease
- +7 more
- Multi-ingredient supplement (PDT-MIS)
- Placebo (PLA)
- (no location specified)
Nov 8, 2023
Lysosomal Storage Diseases, Metachromatic Leukodystrophy Trial in Milan (OTL-200)
Recruiting
- Lysosomal Storage Diseases
- Metachromatic Leukodystrophy
- OTL-200
-
Milan, ItalyOspedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 16, 2022
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (SPK-3006)
Active, not recruiting
- Pompe Disease
- +6 more
- SPK-3006
-
Phoenix, Arizona
- +28 more
Jul 22, 2022
The Natural History of Metachromatic Leukodystrophy
Recruiting
- MLD
- +26 more
- Palliative Care
- Hematopoetic Stem Cell Transplantation
-
Pittsburgh, PennsylvaniaThe Program for the Study of Neurodevelopment in Rare Disorders,
Nov 2, 2021
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2 Trial in Worldwide (Neutralizing Antibody to SPK-3006
Completed
- Pompe Disease
- +5 more
- Neutralizing Antibody to SPK-3006 capsid
-
Phoenix, Arizona
- +17 more
Jul 14, 2021
Fabry Disease, Fabry Disease, Cardiac Variant, Lysosomal Storage Diseases Trial in Wuerzburg (blood sampling
Enrolling by invitation
- Fabry Disease
- +4 more
- blood sampling (alpha-Galactosidase & LysoGb3)
-
Wuerzburg, Bavaria, GermanyUniversity Hospital Wuerzburg
Jul 2, 2021
Complement Activation in the Lysosomal Storage Disorders
Withdrawn
- Fabry Disease
- +3 more
- Complement measurements
-
Melbourne, Victoria, AustraliaThe Royal Melbourne Hospital
Jun 2, 2021
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
Completed
- Lysosomal Storage Diseases
- +2 more
-
Tirana, Albania
- +7 more
May 27, 2021
Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases Trial in Gainesville (Diet and Exercise)
Completed
- Pompe Disease
- +2 more
- Diet and Exercise
-
Gainesville, FloridaUniversity of Florida Clinical Research Center
May 13, 2021
Children With Metachromatic Leukodystrophy
Terminated
- Lipid Metabolism Disorders
- +13 more
-
Torrance, California
- +13 more
Mar 16, 2021
Fabry Disease, Lysosomal Storage Diseases Trial in Worldwide (FLT190)
Unknown status
- Fabry Disease
- Lysosomal Storage Diseases
- FLT190
-
Berlin, Germany
- +4 more
Feb 2, 2021
Fabry Disease, Lysosomal Storage Diseases Trial in London (FLT190)
Recruiting
- Fabry Disease
- Lysosomal Storage Diseases
- FLT190
-
London, United KingdomRoyal Free London
Sep 18, 2020
Lysosomal Storage Disorders Diseases in Minority Groups
Unknown status
- Lysosomal Storage Diseases
- Enzyme assay and molecular sequencing
-
Fairfax, VirginiaLDRTC
Jan 18, 2019
Cellular Pharmacodynamics of Small Molecules in Lysosomal
Unknown status
- Lysosomal Storage Diseases
-
Fairfax, VirginiaLDRTC
Jan 18, 2019
Lysosomal Storage Diseases, Inborn Errors of Metabolism Trial in Durham (ALD-601)
Withdrawn
- Lysosomal Storage Diseases
- Inborn Errors of Metabolism
- ALD-601
-
Durham, North Carolina
- +1 more
Jun 27, 2017