Not applicableLooking for participantsNCT03564561What this trial is testingNatural History of Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II, Adult Assistance Publique - Hôpitaux de Paris 20
Not applicableUnknownNCT02000310What this trial is testingMolecular and Cellular Mechanisms of Lysosomal Storage DiseasesWho this might be right forLysosomal Storage Disorders O & O Alpan LLC 80
Not applicableActive Not RecruitingNCT04848779What this trial is testingA Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)Who this might be right forGlycogen Storage Disease Type II Sanofi 16
Not applicableUnknownNCT04476550What this trial is testingClinical Specimen Collection From Pompe Disease PatientsWho this might be right forPompe Disease Serhat Gumrukcu, MD PhD 12
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 applicableLooking for participantsNCT06539169What this trial is testingFLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesWho this might be right forAlpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more xCures 1,000
Not applicableAvailableNCT04327973What this trial is testingExpanded Access for ATB200/AT2221 for the Treatment of IOPDWho this might be right forPompe Disease Infantile-Onset Amicus Therapeutics
Early research (Phase 1)Ended earlyNCT02185651What this trial is testingA Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated ReactionWho this might be right forPompe DiseaseHypersensitivity Reaction University of Florida 2
Testing effectiveness (Phase 2)Study completedNCT00467935What this trial is testingScotoma Reduction in AMD Patients Treated With RanibizumabWho this might be right forMacular Degeneration Retina Research Foundation 20
Not applicableStudy completedNCT04724213What this trial is testingDevelopment of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe DiseaseWho this might be right forPompe Disease University Children's Hospital, Zurich 101
Large-scale testing (Phase 3)Study completedNCT03729362What this trial is testingA Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseWho this might be right forPompe Disease (Late-onset) Amicus Therapeutics 125
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
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
Testing effectiveness (Phase 2)Study completedNCT02675465What this trial is testingFirst-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221Who this might be right forPompe Disease Amicus Therapeutics 29
Not applicableStudy completedNCT02363153What this trial is testingDiet and Exercise in Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type IILysosomal Storage Diseases University of Florida 2
Post-approval studies (Phase 4)Study completedNCT00701129What this trial is testingAn Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type II Genzyme, a Sanofi Company 4
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
Not applicableEnrolling By InvitationNCT07072676What this trial is testingThe Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Who this might be right forInclusion Body MyositisMyotonic Dystrophy 1Myotonic Dystrophy 2+10 more LMU Klinikum 30
Very early researchStudy completedNCT06109948What this trial is testingStudy to Assess the Safety, Tolerability, PK and PD of ABX1100Who this might be right forHealthyLate Onset Pompe Disease Aro Biotherapeutics 46
Testing effectiveness (Phase 2)Looking for participantsNCT07123155What this trial is testingStudy 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 forPompe Disease Shionogi 45