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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableActive Not RecruitingNCT06052800
What this trial is testing

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

Who this might be right for
Fabry Disease
Sanofi 78
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
Not applicableNo Longer AvailableNCT00962260
What this trial is testing

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer
Testing effectiveness (Phase 2)Study completedNCT02292654
What this trial is testing

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 20
Not applicableUnknownNCT03811496
What this trial is testing

Biomarker Analysis for GBA Associated Parkinson's Disease

Who this might be right for
Parkinson DiseaseGaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100
Not applicableStudy completedNCT03233841
What this trial is testing

Farber Disease Natural History Study

Who this might be right for
Farber DiseaseFarber's DiseaseFarber Lipogranulomatosis+4 more
Sumitomo Pharma Switzerland GmbH 45
Testing effectiveness (Phase 2)WithdrawnNCT03228940
What this trial is testing

Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease

Who this might be right for
Fabry Disease
Resverlogix Corp
Not applicableStudy completedNCT00683189
What this trial is testing

Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
The Cooper Health System 10
Testing effectiveness (Phase 2)Study completedNCT00283959
What this trial is testing

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

Who this might be right for
Fabry Disease
Amicus Therapeutics 4
Testing effectiveness (Phase 2)Study completedNCT02228460
What this trial is testing

Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 11
Testing effectiveness (Phase 2)Temporarily pausedNCT01463215
What this trial is testing

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

Who this might be right for
Type I Gaucher Disease
Exsar Corporation 20
Not applicableUnknownNCT03230591
What this trial is testing

Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy

Who this might be right for
Fabry Disease
Yonsei University 25
Not applicableStudy completedNCT05628259
What this trial is testing

The Effect of Tele-Nursing Based Motivational Interviewing in Individuals With Type 2 Diabetes:RCT

Who this might be right for
Type2 DiabetesSelf EfficacySelf Management+5 more
Pamukkale University 70
Large-scale testing (Phase 3)Study completedNCT03566017
What this trial is testing

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 97
Not applicableStudy completedNCT00001491
What this trial is testing

Analysis of the Nervous System in Patients With Fabry's Disease

Who this might be right for
Fabry's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 325
Post-approval studies (Phase 4)Study completedNCT05054387
What this trial is testing

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 22
Large-scale testing (Phase 3)Study completedNCT00943111
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company 160
Early research (Phase 1)Study completedNCT00001234
What this trial is testing

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 120
Testing effectiveness (Phase 2)Study completedNCT00071877
What this trial is testing

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Large-scale testing (Phase 3)Study completedNCT00925301
What this trial is testing

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 67
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