Lysosomal Storage Diseases: all 123 studies
Page 2 of 2. Open to participants first (16), then studies with results posted, then the rest; newest registration first within each.
- Identification of Undiagnosed Lysosomal Acid Lipase DeficiencyStatus unknownNCT01716728Not applicable
- Gene Therapy for Metachromatic Leukodystrophy (MLD)CompletedNCT01560182Phase 1/2
- An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease PhenotypeCompletedNCT01528917
- Migalastat Food Effect StudyCompletedNCT01489995Phase 1
- Providing Access to Cord Blood Units for TransplantsWithdrawnNCT01479582Phase 2
- Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)WithdrawnNCT01458613
- Characterization of the Patient Population With GalactosialidosisCompletedNCT01416467
- Biomarker for Niemann Pick Type C Disease (BioNPC)WithdrawnNCT01306604
- The Natural History of Mucolipidosis Type IVTerminatedNCT01067742
- Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage DiseasesWithdrawnNCT01003912Phase 1
- A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS ICompletedNCT00852358Not applicable
- A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)TerminatedNCT00787995
- Extension Study of Intrathecal Enzyme Replacement Therapy for MPS ITerminatedNCT00786968Phase 1
- ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesTerminatedNCT00654433Phase 3
- Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 GangliosidosisCompletedNCT00418847Phase 2
- Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)TerminatedNCT00410566Phase 1
- Study to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseCompletedNCT00351156
- Positron Emission Tomography (PET) Imaging in People With Gaucher MutationsCompletedNCT00302146
- Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) ITerminatedNCT00215527Phase 1
- The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control StudyCompletedNCT00071903
- Study of Inborn Errors of Cholesterol Synthesis and Related DisordersTerminatedNCT00046202
- Magnetic Stimulation of the Human Nervous SystemCompletedNCT00001780
- The Classification and Cause of Leukodystrophies of Unknown CauseCompletedNCT00001671