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Results for “Pompe Disease”

179 trials

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Showing 20 of 179 results

Testing effectiveness (Phase 2)Study completedNCT02675465
What this trial is testing

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Who this might be right for
Pompe Disease
Amicus Therapeutics 29
Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 90
Not applicableEnrolling By InvitationNCT06605612
What this trial is testing

Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

Who this might be right for
Inclusion Body MyositisMyotonic DystrophyLimb-girdle and Facioscapulohumeral Muscular Dystrophies+9 more
LMU Klinikum 108
Not applicableStudy completedNCT07015970
What this trial is testing

Real-life Follow-up of the Physical Activity in Type 1 Diabetes Participants Equipped With an Insulin Pump With Hybrid Closed Loop

Who this might be right for
Diabetes Mellitus Type 1Glucose Metabolism DisorderGlycemic Control for Diabetes Mellitus+4 more
ISIS Diabete Service 87
Testing effectiveness (Phase 2)Active Not RecruitingNCT05793307
What this trial is testing

Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug

Who this might be right for
Pompe Disease Infantile-Onset
GeneCradle Inc 16
Not applicableUnknownNCT01409486
What this trial is testing

Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry

Who this might be right for
Pompe Disease
Rambam Health Care Campus 10,000
Not applicableEnded earlyNCT00277979
What this trial is testing

3D Echocardiography Managing Infantile Pompe's Disease

Who this might be right for
Congenital Disorders
Children's Healthcare of Atlanta 2
Testing effectiveness (Phase 2)Active Not RecruitingNCT04174105
What this trial is testing

Gene Transfer Study in Patients With Late Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Astellas Gene Therapies 11
Not applicableStudy completedNCT01773954
What this trial is testing

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

Who this might be right for
Age-Related Macular DegenerationMacular Degeneration
MidAtlantic Retina 40
Post-approval studies (Phase 4)UnknownNCT02405624
What this trial is testing

CPAP for Infantile Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 3
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi ETS 20
Not applicableStudy completedNCT00077662
What this trial is testing

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 61
Not applicableLooking for participantsNCT06557369
What this trial is testing

A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD

Who this might be right for
Intermediate AMD
Oculox Technologies SA 30
Testing effectiveness (Phase 2)WithdrawnNCT04094948
What this trial is testing

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Duke University
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Large-scale testing (Phase 3)Ended earlyNCT01924845
What this trial is testing

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Who this might be right for
Late-onset Pompe Disease
BioMarin Pharmaceutical 24
Not applicableStudy completedNCT02903654
What this trial is testing

Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana

Who this might be right for
Pompe's Disease
Centre Hospitalier de Cayenne 925
Not applicableUnknownNCT04755751
What this trial is testing

Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.

Who this might be right for
Pompe Disease Infantile-Onset
Rambam Health Care Campus 4
Not applicableUnknownNCT03812042
What this trial is testing

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Who this might be right for
Lysosomal Storage Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100,000
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
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