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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Post-approval studies (Phase 4)WithdrawnNCT04143958
What this trial is testing

To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease

Who this might be right for
Fabry Disease
Sanofi
Not applicableEnded earlyNCT03896607
What this trial is testing

Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Who this might be right for
Gaucher DiseaseLiver FibrosisSpleen; Fibrosis
HaEmek Medical Center, Israel 18
Early research (Phase 1)Study completedNCT00337636
What this trial is testing

Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

Who this might be right for
Neuronal Ceroid Lipofuscinosis
StemCells, Inc. 6
Not applicableStudy completedNCT05359276
What this trial is testing

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)
Sanofi 40
Post-approval studies (Phase 4)Study completedNCT04120506
What this trial is testing

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 15
Not applicableUnknownNCT02520934
What this trial is testing

Miglustat on Gaucher Disease Type IIIB

Who this might be right for
Gaucher Disease
National Taiwan University Hospital 19
Not applicableStudy completedNCT04721366
What this trial is testing

Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

Who this might be right for
Gaucher Disease
Takeda 11
Not applicableLooking for participantsNCT06539624
What this trial is testing

Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease

Who this might be right for
Fabry Disease
The Children's Hospital of Zhejiang University School of Medicine 12
Not applicableStudy completedNCT00244582
What this trial is testing

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

Who this might be right for
Type 2 Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 1
Not applicableLooking for participantsNCT02993796
What this trial is testing

Krabbe Disease Global Patient Registry

Who this might be right for
Krabbe Disease
State University of New York at Buffalo 60
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Not applicableAvailableNCT01476163
What this trial is testing

Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics
Large-scale testing (Phase 3)Not Yet RecruitingNCT07778667
What this trial is testing

Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease

Who this might be right for
Fabry Disease
Idorsia Pharmaceuticals Ltd. 16
Not applicableWithdrawnNCT04965467
What this trial is testing

Fabry Aim Children Early (ACE) Project

Who this might be right for
Fabry Disease
Children's Hospital of Fudan University
Not applicableNot Yet RecruitingNCT07172672
What this trial is testing

The TiMeD-T1D Project

Who this might be right for
Postprandial Lipid MetabolismType 1 Diabetes (T1D)
University of Aarhus 10
Not applicableStudy completedNCT00531037
What this trial is testing

Natural Evolution of AV Conduction Disorders in Patients Implanted With Cardiac Pacemakers

Who this might be right for
Patients Implanted With a Pacemaker
LivaNova 1,440
Not applicableUnknownNCT04324775
What this trial is testing

Comparison of Acute Effect of Different Manual Techniques on Muscle Fascia Stiffness

Who this might be right for
Manual Lymphatic DrainageSwedish MassageMuscle Fascia Stiffness
Marmara University 30
Not applicableAvailableNCT07675031
What this trial is testing

Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).

Who this might be right for
Type 3 Gaucher Disease
Sanofi
Large-scale testing (Phase 3)Ended earlyNCT04395339
What this trial is testing

GM1 Prophylaxis for WBRT Related Cognitive Dysfunction

Who this might be right for
Brain MetastasesRadiotherapy Side EffectCognitive Impairment+2 more
Sun Yat-sen University 51
Not applicableEnded earlyNCT01698229
What this trial is testing

Collection of Cerebrospinal Fluid in Healthy Children

Who this might be right for
Late Infantile Neuronal Ceroid LipofuscinosisBatten Disease
Weill Medical College of Cornell University 32
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