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Results for “Lysosomal Storage Disease”

1,037 trials

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Showing 20 of 1,037 results

Not applicableLooking for participantsNCT06610019
What this trial is testing

Cardiovascular Multimodality Imaging Study

Who this might be right for
Non-ischemic CardiomyopathyCardiomyopathiesHypertrophic Cardiomyopathy+7 more
Montefiore Medical Center 5,000
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Post-approval studies (Phase 4)Study completedNCT00144781
What this trial is testing

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 34
Not applicableEnded earlyNCT02095015
What this trial is testing

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 159
Early research (Phase 1)Ended earlyNCT00875160
What this trial is testing

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

Who this might be right for
Type 1 Gaucher Disease
Amicus Therapeutics 1
Early research (Phase 1)Study completedNCT01730482
What this trial is testing

Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)

Who this might be right for
Fabry Disease
Amicus Therapeutics 6
Not applicableStudy completedNCT00484549
What this trial is testing

Fabry : National Initiative of Screening

Who this might be right for
Ischemic Stroke Hospitalized
University Hospital, Clermont-Ferrand 889
Very early researchLooking for participantsNCT06207552
What this trial is testing

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Who this might be right for
Fabry Disease
Children's Hospital of Fudan University 6
Not applicableStudy completedNCT00055016
What this trial is testing

Registry of Fabry Disease - A Multicenter Observational Study

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
Not applicableStudy completedNCT00010426
What this trial is testing

Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

Who this might be right for
Cystinosis
FDA Office of Orphan Products Development 30
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
Testing effectiveness (Phase 2)Study completedNCT00283933
What this trial is testing

A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 5
Not applicableUnknownNCT06169358
What this trial is testing

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy

Who this might be right for
Hypertrophic CardiomyopathyLeft Ventricular HypertrophyFabry Disease
Qilu Hospital of Shandong University 627
Early research (Phase 1)Study completedNCT02262338
What this trial is testing

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
ArmaGen, Inc 6
Not applicableEnrolling By InvitationNCT05146830
What this trial is testing

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Who this might be right for
Cystinosis
Stephanie Cherqui 50
Not applicableActive Not RecruitingNCT04929002
What this trial is testing

Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

Who this might be right for
Glycogen Storage DiseaseMcArdle DiseasePompe Disease (Late-onset)
Rigshospitalet, Denmark 50
Testing effectiveness (Phase 2)Study completedNCT04455230
What this trial is testing

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Who this might be right for
Fabry DiseaseLysosomal Storage Diseases
Spur Therapeutics 3
Large-scale testing (Phase 3)Ended earlyNCT04840667
What this trial is testing

Replagal in Treatment-naïve Adults With Fabry Disease

Who this might be right for
Fabry Disease
Shire 17
Not applicableStudy completedNCT01355146
What this trial is testing

Home Therapy With Replagal in Fabry Disease

Who this might be right for
Fabry Disease
Shire 127
Large-scale testing (Phase 3)Looking for participantsNCT02716246
What this trial is testing

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 36
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