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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00283933
What this trial is testing

A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 5
Not applicableStudy completedNCT04628364
What this trial is testing

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

Who this might be right for
Metachromatic Leukodystrophy
National Organization for Rare Disorders 21
Not applicableStudy completedNCT02416661
What this trial is testing

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

Who this might be right for
Lysosomal Storage DiseasesGaucher DiseaseSphingolipidoses
CENTOGENE GmbH Rostock 299
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
Not applicableUnknownNCT01695161
What this trial is testing

Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.

Who this might be right for
MucopolysaccharidosisFabry Disease
Johannes Gutenberg University Mainz 75
Not applicableNo Longer AvailableNCT02021266
What this trial is testing

Single Patient Expanded Access Protocol: Metabolic Boost

Who this might be right for
Metachromatic Leukodystrophy
Talaris Therapeutics Inc.
Not applicableNot Yet RecruitingNCT05669729
What this trial is testing

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

Who this might be right for
Gaucher Disease
Takeda 60
Not applicableUnknownNCT04184986
What this trial is testing

Screening of Fabry Disease in Patients With GI Symptoms

Who this might be right for
Fabry Disease
General University Hospital, Prague 200
Not applicableUnknownNCT03875105
What this trial is testing

Daily Living Activities and Exercise Capacity in Lipid Storage Diseases

Who this might be right for
Lipid Storage Disease
Istanbul University 25
Not applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60
Large-scale testing (Phase 3)Study completedNCT00319046
What this trial is testing

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Actelion 42
Large-scale testing (Phase 3)Study completedNCT00864851
What this trial is testing

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

Who this might be right for
Fabry Disease
Shire 44
Testing effectiveness (Phase 2)Study completedNCT00517153
What this trial is testing

Miglustat in Niemann-Pick Type C Disease

Who this might be right for
Niemann-Pick Type C Disease
Actelion 29
Not applicableWithdrawnNCT02383641
What this trial is testing

Biomarker for Wolman Disease (BioWolman)

Who this might be right for
Acid Lipase DeficiencyAcid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeCholesterol Ester Storage Disease
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Ended earlyNCT04455230
What this trial is testing

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Who this might be right for
Fabry DiseaseLysosomal Storage Diseases
Spur Therapeutics 3
Not applicableStudy completedNCT01295008
What this trial is testing

Androgenetic Alopecia in Fabry Disease

Who this might be right for
Fabry Disease
Baylor Research Institute 107
Large-scale testing (Phase 3)Study completedNCT02004691
What this trial is testing

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 36
Not applicableLooking for participantsNCT06669949
What this trial is testing

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

Who this might be right for
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
University of California, San Francisco 28
Not applicableLooking for participantsNCT06813443
What this trial is testing

Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation

Who this might be right for
CardiomyopathiesAmyloidosis CardiacFabry Disease+14 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 700
Not applicableEnrolling By InvitationNCT03862274
What this trial is testing

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Who this might be right for
Batten DiseaseCLN2Neuronal Ceroid-Lipofuscinoses
Jessica Scherr 30
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