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
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
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
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
Not applicableUnknownNCT01914536What this trial is testingA Natural History Study of Adult Onset Pompe Disease Using Muscle MRIWho this might be right forPompe Disease Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 30
Not applicableLooking for participantsNCT01665326What this trial is testingDetermination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseWho this might be right forPompe Disease Duke University 400
Post-approval studies (Phase 4)Ended earlyNCT01597596What this trial is testingA Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe DiseaseWho this might be right forPompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more Genzyme, a Sanofi Company 4
Testing effectiveness (Phase 2)Study completedNCT00025896What this trial is testingSafety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe DiseaseWho this might be right forPompe DiseaseGlycogen Storage Disease Type IIAcid Maltase Deficiency Disease+1 more Genzyme, a Sanofi Company 8
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
Early research (Phase 1)Looking for participantsNCT07354724What this trial is testingEvaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseWho this might be right forLate-onset Pompe Disease Denali Therapeutics Inc. 32
Post-approval studies (Phase 4)Study completedNCT00483379What this trial is testingHigh Dose or High Dose Frequency Study of Alglucosidase AlfaWho this might be right forPompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 13
Not applicableStudy completedNCT03893240What this trial is testingNeutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe DiseaseWho this might be right forPompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2+3 more Spark Therapeutics, Inc. 61
Not applicableApproved For MarketingNCT00074919What this trial is testingExpanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type IIGlycogenosis 2 Genzyme, a Sanofi Company
Testing effectiveness (Phase 2)UnknownNCT06130228What this trial is testingNutritional Therapy in Late-onset Pompe DiseaseWho this might be right forPompe DiseaseMuscle LossObesity+5 more McMaster University 28
Testing effectiveness (Phase 2)WithdrawnNCT01656590What this trial is testingHigh Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe DiseaseWho this might be right forGlycogen Storage Disease Type II Columbia University
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)Study completedNCT00763932What this trial is testingExtension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) StudiesWho this might be right forPompe Disease Infantile-OnsetGlycogen Storage Disease Type II Genzyme, a Sanofi Company 7
Post-approval studies (Phase 4)Ended earlyNCT00701701What this trial is testingImmune Tolerance Induction StudyWho this might be right forPompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency Genzyme, a Sanofi Company 4
Not applicableStudy completedNCT00566878What this trial is testingPompe Lactation Sub-RegistryWho this might be right forGlycogen Storage DiseasePompe Disease Genzyme, a Sanofi Company 2