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Results for “Storage Disease”

1,384 trials

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Showing 20 of 1,384 results

Not applicableStudy completedNCT01632111
What this trial is testing

Pulmonary Involvement in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University of Zurich 110
Testing effectiveness (Phase 2)Looking for participantsNCT06940011
What this trial is testing

Endocalyx in Treatment Resistent Hypertension

Who this might be right for
Treatment Resistant Hypertension
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 64
Post-approval studies (Phase 4)Study completedNCT05134571
What this trial is testing

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

Who this might be right for
Mucopolysaccharidosis I
Genzyme, a Sanofi Company 12
Not applicableStudy completedNCT01416467
What this trial is testing

Characterization of the Patient Population With Galactosialidosis

Who this might be right for
Galactosialidosis
St. Jude Children's Research Hospital 3
Large-scale testing (Phase 3)Active Not RecruitingNCT04910776
What this trial is testing

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II
Sanofi 17
Post-approval studies (Phase 4)Study completedNCT00097890
What this trial is testing

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableStudy completedNCT01961518
What this trial is testing

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

Who this might be right for
Morquio Syndrome AMaroteaux Lamy SyndromeMPS IVA+1 more
Greenwood Genetic Center 17
Large-scale testing (Phase 3)Not Yet RecruitingNCT07579910
What this trial is testing

Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)

Who this might be right for
MPS IIIB
Spruce Biosciences 14
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Not applicableStudy completedNCT04081272
What this trial is testing

Effect of G6PD Deficiency on Red Blood Cell Storage

Who this might be right for
G6PD Deficiency
Columbia University 40
Not applicableEnded earlyNCT02345421
What this trial is testing

Identify and Characterize LAL-D Patients in High-risk Populations

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 640
Not applicableWithdrawnNCT02778295
What this trial is testing

Biomarker for Patients With Fabry Disease (BioFabry)

Who this might be right for
AngiokeratomasChronic Kidney DiseaseOcular Abnormalities+1 more
CENTOGENE GmbH Rostock
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
Large-scale testing (Phase 3)Looking for participantsNCT05955001
What this trial is testing

Efficacy of Tadalafil (5mg) For Treatment of Early Storage Symptoms and Erectile Dysfunction After Endoscopic Enucleation of Prostate

Who this might be right for
Erectile DysfunctionLower Urinary Tract Symptoms
Mansoura University 110
Testing effectiveness (Phase 2)Active Not RecruitingNCT04201405
What this trial is testing

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis Type IIIA
University of Manchester 5
Large-scale testing (Phase 3)Study completedNCT02782741
What this trial is testing

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II;Pompe's Disease
Genzyme, a Sanofi Company 101
Not applicableUnknownNCT02676570
What this trial is testing

Validation of Pathologic Diagnosis of Invasive Fungal Infection by Molecular Method

Who this might be right for
Mycoses
Seoul St. Mary's Hospital 50
Testing effectiveness (Phase 2)Ended earlyNCT03746587
What this trial is testing

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
ZevraDenmark 39
Testing effectiveness (Phase 2)UnknownNCT00004293
What this trial is testing

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Who this might be right for
Gaucher's Disease
National Center for Research Resources (NCRR) 24
Testing effectiveness (Phase 2)Study completedNCT02489344
What this trial is testing

Evaluation of the Long-term Safety, Pharmacodynamics, 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 8
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