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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT06143904
What this trial is testing

Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants

Who this might be right for
Gaucher's Disease Type I
Sanofi 10
Early research (Phase 1)Study completedNCT01747980
What this trial is testing

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Protalix 16
Not applicableStudy completedNCT05409846
What this trial is testing

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

Who this might be right for
Fabry Disease
Universidade do Porto 409
Not applicableUnknownNCT02605603
What this trial is testing

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Who this might be right for
Gaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 30
Testing effectiveness (Phase 2)Study completedNCT05758922
What this trial is testing

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Disease, Type C
Azafaros B.V. 13
Not applicableApproved For MarketingNCT00954460
What this trial is testing

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire
Not applicableUnknownNCT04847713
What this trial is testing

Early Detection and Follow-Up of Patients With Fabry's Disease

Who this might be right for
Fabry's Disease
Hospital Clinico Universitario de Santiago 100
Not applicableUnknownNCT05413876
What this trial is testing

Fabry Exercise Intolerance Study

Who this might be right for
Fabry DiseaseFabry Disease, Cardiac Variant
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 30
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07603050
What this trial is testing

A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

Who this might be right for
Gaucher Disease Type 3
Shanghai Vitalgen BioPharma Co., Ltd. 12
Not applicableActive Not RecruitingNCT06488261
What this trial is testing

Long Term Follow-Up for Safety of AVR-RD-02

Who this might be right for
Gaucher Disease
John Bernat 2
Large-scale testing (Phase 3)Ended earlyNCT03822013
What this trial is testing

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Who this might be right for
GM2 GangliosidosisSupportive Care
Tehran University of Medical Sciences 30
Early research (Phase 1)Study completedNCT00258778
What this trial is testing

Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)

Who this might be right for
Gaucher Disease
Protalix 6
Post-approval studies (Phase 4)Study completedNCT04718779
What this trial is testing

Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Who this might be right for
Gaucher Disease
Takeda 4
Large-scale testing (Phase 3)Study completedNCT01298141
What this trial is testing

Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

Who this might be right for
Fabry Disease
Shire 171
Post-approval studies (Phase 4)Study completedNCT00081497
What this trial is testing

The Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 67
Testing effectiveness (Phase 2)Study completedNCT00446550
What this trial is testing

Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 19
Not applicableNot Yet RecruitingNCT07173010
What this trial is testing

Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital

Who this might be right for
MucopolysaccharidosesProgressive Pseudorheumatoid DysplasiaFarber Disease+3 more
Assiut University 35
Not applicableStudy completedNCT00001289
What this trial is testing

Effects of Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 70
Large-scale testing (Phase 3)Study completedNCT01614574
What this trial is testing

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 6
Not applicableStudy completedNCT04470713
What this trial is testing

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

Who this might be right for
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more
Idorsia Pharmaceuticals Ltd. 226
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