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Results for “Pompe Disease Late-Onset”

66 trials

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Showing 20 of 66 results

Not applicableStudy completedNCT00074932
What this trial is testing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIGlycogenosis 2
Genzyme, a Sanofi Company 9
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349
What this trial is testing

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more
Spark Therapeutics, Inc. 4
Testing effectiveness (Phase 2)UnknownNCT06130228
What this trial is testing

Nutritional Therapy in Late-onset Pompe Disease

Who this might be right for
Pompe DiseaseMuscle LossObesity+5 more
McMaster University 28
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
Not applicableLooking for participantsNCT00231400
What this trial is testing

Pompe Disease Registry Protocol

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease
Genzyme, a Sanofi Company 2,000
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
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
Early research (Phase 1)Study completedNCT02240407
What this trial is testing

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 2
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Not applicableUnknownNCT04981210
What this trial is testing

High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics

Who this might be right for
Pompe Disease
National Taiwan University Hospital 1,500
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
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
Not applicableStudy completedNCT05431127
What this trial is testing

High Dose Inspiratory Muscle Training in LOPD

Who this might be right for
Late-Onset Pompe DiseaseLysosomal Disease
Duke University 34
Post-approval studies (Phase 4)Study completedNCT00455195
What this trial is testing

Late-Onset Treatment Study Extension Protocol

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD-II)Glycogenesis Type II+1 more
Genzyme, a Sanofi Company 81
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
Testing effectiveness (Phase 2)Study completedNCT00765414
What this trial is testing

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Who this might be right for
Pompe Disease Late-OnsetGlycogen Storage Disease Type II GSD II
Genzyme, a Sanofi Company 1
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
Large-scale testing (Phase 3)Study completedNCT00268944
What this trial is testing

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

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 5
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 applicableStudy completedNCT02801539
What this trial is testing

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

Who this might be right for
Glycogen Storage Disease Type II
Duke University 22
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