Early research (Phase 1)Not Yet RecruitingNCT07787650What this trial is testingS-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)Who this might be right forPompe Disease Shionogi 15
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Large-scale testing (Phase 3)Study completedNCT00125879What this trial is testingExtension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602Who this might be right forGlycogen Storage Disease Type II Genzyme, a Sanofi Company 16
Not applicableUnknownNCT05272969What this trial is testingPompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseaseWho this might be right forPompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more LMU Klinikum 95
Not applicableActive Not RecruitingNCT03694561What this trial is testingDeveloping a Management Approach for Patients With "Late-Onset" Pompe DiseaseWho this might be right forPompe DiseasePompe Disease (Late-onset)GAA Deficiency Duke University 20
Not applicableActive Not RecruitingNCT04929002What this trial is testingCarbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage DiseasesWho this might be right forGlycogen Storage DiseaseMcArdle DiseasePompe Disease (Late-onset) Rigshospitalet, Denmark 50
Post-approval studies (Phase 4)Study completedNCT00455195What this trial is testingLate-Onset Treatment Study Extension ProtocolWho this might be right forPompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD-II)Glycogenesis Type II+1 more Genzyme, a Sanofi Company 81
Testing effectiveness (Phase 2)Study completedNCT00250939What this trial is testingRhGAA in Patients With Late-Onset Pompe DiseaseWho this might be right forPompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 5
Very early researchNot Yet RecruitingNCT06178432What this trial is testingEvaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)Who this might be right forPompe Disease (Late-onset) Huashan Hospital 6
Not applicableEnded earlyNCT03347253What this trial is testingSTRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERTWho this might be right forLate-onset Pompe Disease Amicus Therapeutics 12
Testing effectiveness (Phase 2)Ended earlyNCT02898753What this trial is testingVAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe DiseaseWho this might be right forPompe Disease Valerion Therapeutics, LLC 12
Large-scale testing (Phase 3)Ended earlyNCT01924845What this trial is testingBMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)Who this might be right forLate-onset Pompe Disease BioMarin Pharmaceutical 24
Early research (Phase 1)Study completedNCT01898364What this trial is testingSafety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.Who this might be right forPompe DiseaseGlycogen Storage Disease Type II (GSD II)Acid Maltase Deficiency Genzyme, a Sanofi Company 24
Not applicableLooking for participantsNCT00231400What this trial is testingPompe Disease Registry ProtocolWho this might be right forGlycogen Storage Disease Type IIPompe Disease Genzyme, a Sanofi Company 2,000
Large-scale testing (Phase 3)Study completedNCT00268944What this trial is testingSafety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportWho this might be right forPompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 5
Not applicableStudy completedNCT00074932What this trial is testingExpanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type IIGlycogenosis 2 Genzyme, a Sanofi Company 9
Testing effectiveness (Phase 2)Active Not RecruitingNCT04093349What this trial is testingA Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)Who this might be right forPompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+4 more Spark Therapeutics, Inc. 4
Not applicableStudy completedNCT05431127What this trial is testingHigh Dose Inspiratory Muscle Training in LOPDWho this might be right forLate-Onset Pompe DiseaseLysosomal Disease Duke University 34
Not applicableStudy completedNCT00077662What this trial is testingA Prospective, Observational Study in Patients With Late-Onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II Genzyme, a Sanofi Company 61
Not applicableEnded earlyNCT02221362What this trial is testingA Prospective, Noninterventional, Observational Study of Late-Onset Pompe DiseaseWho this might be right forLate-onset Pompe Patients Untreated or Treated With rhGAA BioMarin Pharmaceutical 30