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Results for “Lysosomal Storage Diseases”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Ended earlyNCT03879655
What this trial is testing

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 2
Not applicableStudy completedNCT05106764
What this trial is testing

Early Detection of Fabry Disease

Who this might be right for
Fabry Disease
Takeda 50
Post-approval studies (Phase 4)Study completedNCT00779493
What this trial is testing

Curcumin (Tumeric) in the Treatment of Irritable Bowel Syndrome: A Randomized-Controlled Trial

Who this might be right for
Irritable Bowel Syndrome
Kaiser Permanente 17
Not applicableUnknownNCT05816993
What this trial is testing

Assessment of Neurological Manifestations in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Assiut University 45
Early research (Phase 1)Study completedNCT04539340
What this trial is testing

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 20
Not applicableLooking for participantsNCT04613089
What this trial is testing

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Who this might be right for
Neuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more
Universitätsklinikum Hamburg-Eppendorf 500
Not applicableUnknownNCT02761421
What this trial is testing

Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 15
Not applicableUnknownNCT02708784
What this trial is testing

An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-

Who this might be right for
Glycogen Storage Disease Type 2Dystrophia Myotonica
University of Aarhus 60
Post-approval studies (Phase 4)Ended earlyNCT01597596
What this trial is testing

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more
Genzyme, a Sanofi Company 4
Not applicableUnknownNCT01482494
What this trial is testing

Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia

Who this might be right for
Pompe Disease
Jordi Perez Lopez 50
Not applicableUnknownNCT01716728
What this trial is testing

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage Disease
Massachusetts General Hospital 60
Not applicableStudy completedNCT05648851
What this trial is testing

A Natural History Study of Sanfilippo Syndrome Type D

Who this might be right for
Sanfilippo Syndrome Type D
Phoenix Nest 10
Testing effectiveness (Phase 2)Study completedNCT02437253
What this trial is testing

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
Not applicableStudy completedNCT04997772
What this trial is testing

Digital Health Platform Customized for Patients With Gaucher Disease

Who this might be right for
Gaucher DiseasePatient Reported Outcome
Shoshana Vilk 80
Early research (Phase 1)Study completedNCT01489995
What this trial is testing

Migalastat Food Effect Study

Who this might be right for
Fabry Disease
Amicus Therapeutics 20
Not applicableEnded earlyNCT01963650
What this trial is testing

Natural History Study of Children With Metachromatic Leukodystrophy

Who this might be right for
Lipid Metabolism DisordersMetachromatic Leukodystrophy (MLD)Nervous System Diseases+11 more
Shire 1
Not applicableStudy completedNCT05186324
What this trial is testing

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 23
Large-scale testing (Phase 3)Active Not RecruitingNCT06149403
What this trial is testing

Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Who this might be right for
MPS-IH (Hurler Syndrome)
Orchard Therapeutics 41
Not applicableStudy completedNCT03838237
What this trial is testing

Effect of Migalastat on Cardiac Involvement in Fabry Disease

Who this might be right for
Fabry DiseaseHeart Diseases
Ospedale San Donato 18
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
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