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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableUnknownNCT02469181
What this trial is testing

Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)

Who this might be right for
Fabry's Disease
Yonsei University 20
Not applicableLooking for participantsNCT03240653
What this trial is testing

Gaucherite - A Study to Stratify Gaucher Disease

Who this might be right for
Gaucher Disease, Type IGaucher Disease, Type III
Cambridge University Hospitals NHS Foundation Trust 250
Early research (Phase 1)UnknownNCT03893071
What this trial is testing

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 12
Not applicableStudy completedNCT02450604
What this trial is testing

Prevalence of Fabry's Disease in a Population of Patients With Chronic Pain

Who this might be right for
Fabry's DiseaseChronic Pain
University Hospital, Bordeaux 776
Not applicableStudy completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
Large-scale testing (Phase 3)WithdrawnNCT01161914
What this trial is testing

The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease

Who this might be right for
Gaucher Disease
ISU Abxis Co., Ltd.
Early research (Phase 1)Active Not RecruitingNCT06517914
What this trial is testing

A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants

Who this might be right for
Healthy Participants
Yuhan Corporation 94
Not applicableStudy completedNCT01178164
What this trial is testing

Prevalence of Fabry's Disease in a Population of Patients With Chronic Pains

Who this might be right for
PainFabry's Disease
University Hospital, Bordeaux 137
Not applicableLooking for participantsNCT06036693
What this trial is testing

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more
Institut National de la Santé Et de la Recherche Médicale, France 1,000
Not applicableStudy completedNCT04310163
What this trial is testing

Interviews and Video Capture in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 25
Large-scale testing (Phase 3)Study completedNCT06211478
What this trial is testing

Role of Vitamin E in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Ain Shams University 40
Not applicableStudy completedNCT06935578
What this trial is testing

RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascular Diseases (ALIGNED)

Who this might be right for
CADASILCADASIL (Diagnosis)Moya Moya Disease+5 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 500
Not applicableStudy completedNCT01358188
What this trial is testing

Exploration of Immunity in Gaucher Disease

Who this might be right for
Gaucher Disease
O & O Alpan LLC 33
Not applicableEnded earlyNCT04644549
What this trial is testing

Natural History Study of Batten Disease

Who this might be right for
Batten DiseaseNeuronal Ceroid Lipofuscinosis CLN6Neuronal Ceroid Lipofuscinosis CLN3
Emily de los Reyes 10
Not applicableActive Not RecruitingNCT04476862
What this trial is testing

Cerliponase Alfa Observational Study in the US

Who this might be right for
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
BioMarin Pharmaceutical 35
Testing effectiveness (Phase 2)Study completedNCT00304512
What this trial is testing

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 9
Not applicableLooking for participantsNCT06906367
What this trial is testing

Patients With Fabry Disease (US Specific)

Who this might be right for
Fabry Disease
Amicus Therapeutics 450
Not applicableEnrolling By InvitationNCT06880250
What this trial is testing

Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease

Who this might be right for
Fabry Disease
NPO Petrovax 100
Not applicableStudy completedNCT04084301
What this trial is testing

Impact of Cardiopulmonary Bypass Flow on Renal Oxygenation, Blood Flow and Tubular Injury

Who this might be right for
Extracorporeal Circulation; ComplicationsRenal FailureCirculation Disorder+4 more
Sahlgrenska University Hospital 100
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
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