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Results for “Late Onset Pompe Disease”

65 trials

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Showing 20 of 65 results

Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Not applicableUnknownNCT05272969
What this trial is testing

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more
LMU Klinikum 95
Not applicableStudy completedNCT02654886
What this trial is testing

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

Who this might be right for
Pompe DiseaseMuscle Weakness
University of California, Irvine 10
Not applicableStudy completedNCT00731081
What this trial is testing

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 8
Not applicableStudy completedNCT02354664
What this trial is testing

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 14
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
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
Post-approval studies (Phase 4)Study completedNCT04676373
What this trial is testing

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

Who this might be right for
Pompe's Disease
Genzyme, a Sanofi Company 41
Not applicableLooking for participantsNCT05989620
What this trial is testing

Long-Term Development of Muscular Dystrophy Outcome Assessments

Who this might be right for
LGMD1BLGMD1CLGMD1D+26 more
Virginia Commonwealth University 1,000
Not applicableApproved For MarketingNCT00520143
What this trial is testing

Alglucosidase Alfa Temporary Access Program

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-Onset)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company
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 applicableStudy completedNCT04120168
What this trial is testing

Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease

Who this might be right for
Duchenne Muscular DystrophyPompe Disease (Late-onset)
Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition 590
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
Post-approval studies (Phase 4)Not Yet RecruitingNCT06575829
What this trial is testing

Treatment Frequency Reduction in Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type II+1 more
Erasmus Medical Center 10
Testing effectiveness (Phase 2)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
Not applicableStudy completedNCT02824068
What this trial is testing

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

Who this might be right for
Pompe Disease
LMU Klinikum 112
Early research (Phase 1)Not Yet RecruitingNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Not applicableActive Not RecruitingNCT03694561
What this trial is testing

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)GAA Deficiency
Duke University 20
Very early researchActive Not RecruitingNCT06109948
What this trial is testing

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

Who this might be right for
HealthyLate Onset Pompe Disease
Aro Biotherapeutics 46
Not applicableUnknownNCT03045042
What this trial is testing

Search for Serum/Plasma Biomarkers in Pompe's Disease

Who this might be right for
Late Onset Pompe DiseasePompe Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 35
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