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Results for “Glycogen Storage Disease Type II (Late-onset Pompe Disease)”

70 trials

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Showing 20 of 70 results

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 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 applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
Testing effectiveness (Phase 2)Study completedNCT01230801
What this trial is testing

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
BioMarin Pharmaceutical 22
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
Early research (Phase 1)Study completedNCT03533673
What this trial is testing

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
AskBio Inc 7
Testing effectiveness (Phase 2)Looking for participantsNCT06391736
What this trial is testing

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

Who this might be right for
Pompe Disease (Late-onset)
GeneCradle Inc 33
Early research (Phase 1)Study completedNCT01898364
What this trial is testing

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD II)Acid Maltase Deficiency
Genzyme, a Sanofi Company 24
Large-scale testing (Phase 3)Study completedNCT04138277
What this trial is testing

Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 119
Not applicableEnded earlyNCT03347253
What this trial is testing

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Who this might be right for
Late-onset Pompe Disease
Amicus Therapeutics 12
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
Very early researchStudy completedNCT06109948
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
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
Not applicableStudy completedNCT07685314
What this trial is testing

LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS

Who this might be right for
Late-Onset Pompe Disease
Hospitales Universitarios Virgen del Rocío 477
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
Not applicableStudy completedNCT02765828
What this trial is testing

Identification of Tongue Involvement in Late-Onset Pompe Disease

Who this might be right for
MyopathyNeuropathyGlycogen Storage Disease Type II (Late-onset Pompe Disease)
Duke University 73
Not applicableEnded earlyNCT02191917
What this trial is testing

Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Who this might be right for
Late-onset Pompe Disease
BioMarin Pharmaceutical 8
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
Early research (Phase 1)Looking for participantsNCT07354724
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
Large-scale testing (Phase 3)Study completedNCT03729362
What this trial is testing

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 125
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