Glycogen Storage Disease Type II: all 152 studies

Page 1 of 2. Open to participants first (31), then studies with results posted, then the rest; newest registration first within each.

• MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)Not yet recruitingNCT07892651
• Pompe Disease RWE Long-term Monitoring ProgramRecruitingNCT07834320
• A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)Not yet recruitingNCT07787650Phase 1
• An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)Not yet recruitingNCT07750990Phase 2
• Phrenic Nerve and Diaphragm Electrophysiology in Pompe DiseaseRecruitingNCT07664930
• Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseRecruitingNCT07478172Not applicable
• A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseRecruitingNCT07354724Phase 1
• A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)RecruitingNCT07282847Phase 1/2
• Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)RecruitingNCT07123155Phase 2
• The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Enrolling by invitationNCT07072676Not applicable
• Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle DiseasesRecruitingNCT06833489Not applicable
• China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDRecruitingNCT06666413Phase 4
• Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular DisordersEnrolling by invitationNCT06605612
• Treatment Frequency Reduction in Pompe DiseaseNot yet recruitingNCT06575829Phase 4
• FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesRecruitingNCT06539169
• Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy DrugRecruitingNCT06391736Phase 1/2
• Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)Not yet recruitingNCT06178432Early Phase 1
• Nutritional Therapy in Late-onset Pompe DiseaseNot yet recruitingNCT06130228Phase 2
• A Global Prospective Observational Registry of Patients With Pompe DiseaseRecruitingNCT06121011
• Inspiratory Muscle Training (IMT) in Adult People With Pompe DiseaseRecruitingNCT05951790Not applicable
• Avalglucosidase Alfa Pregnancy StudyRecruitingNCT05734521
• Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe DiseaseRecruitingNCT05567627Not applicable
• Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseaseRecruitingNCT05272969
• Higher Dose of Alglucosidase Alpha for Pompe DiseaseNot yet recruitingNCT05017402
• Cognitive and Neurological Pathologies in Pompe DiseaseRecruitingNCT04639336
• Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseAvailableNCT03865836
• Natural History of Pompe DiseaseRecruitingNCT03564561
• A Long-term Study for the Outcome of Pompe DiseaseRecruitingNCT02399748
• Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseRecruitingNCT01665326
• Pompe Pregnancy Sub-RegistryRecruitingNCT00567073
• Pompe Disease Registry ProtocolRecruitingNCT00231400
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)CompletedNCT04138277Phase 3Results posted
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe DiseaseCompletedNCT03729362Phase 3Results posted
• Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseCompletedNCT02782741Phase 3Results posted
• First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221CompletedNCT02675465Phase 1/2Results posted
• Dynamic Respiratory Muscle Function in Late-Onset Pompe DiseaseCompletedNCT02354664Results posted
• Response to Diaphragmatic Pacing in Subjects With Pompe DiseaseCompletedNCT02354651Results posted
• A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)TerminatedNCT02191917Results posted
• Avalglucosidase Alfa Extension StudyCompletedNCT02032524Phase 2Results posted
• Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement TherapyCompletedNCT01942590Phase 1/2Results posted
• BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)TerminatedNCT01924845Phase 3Results posted
• Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe DiseaseCompletedNCT01885936Phase 1/2Results posted
• A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe DiseaseTerminatedNCT01597596Phase 4Results posted
• A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe DiseaseTerminatedNCT01526785Phase 4Results posted
• Extension Study for Patients Who Have Participated in a BMN 701 StudyTerminatedNCT01435772Phase 2Results posted
• Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe DiseaseCompletedNCT01410890Phase 4Results posted
• Drug-drug Interaction StudyCompletedNCT01380743Phase 2Results posted
• Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase AlfaCompletedNCT01288027Phase 4Results posted
• Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe DiseaseCompletedNCT01230801Phase 1/2Results posted
• Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe DiseaseCompletedNCT00976352Phase 1/2Results posted
• Immune Tolerance Induction StudyTerminatedNCT00701701Phase 4Results posted
• An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseCompletedNCT00701129Phase 4Results posted
• Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe DiseaseTerminatedNCT00688597Phase 2Results posted
• Growth and Development Study of Alglucosidase AlfaCompletedNCT00486889Phase 4Results posted
• High Dose or High Dose Frequency Study of Alglucosidase AlfaCompletedNCT00483379Phase 4Results posted
• Late-Onset Treatment Study Extension ProtocolCompletedNCT00455195Phase 4Results posted
• A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe DiseaseCompletedNCT00158600Phase 3Results posted
• LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONSCompletedNCT07685314
• Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 YearsActive, not recruitingNCT07652814Phase 4
• A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe DiseaseActive, not recruitingNCT06150820Not applicable
• Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.Active, not recruitingNCT05943678
• Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
• ERT in Pompe Disease: Elucidation of Molecular Structures Contributing to Enzyme Uptake and ImmunoreactivityStatus unknownNCT05448131
• Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)Active, not recruitingNCT05164055Phase 4
• Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)Status unknownNCT05092230
• MSOT in Pompe DiseaseCompletedNCT05083806Not applicable
• A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care SettingCompletedNCT05073783
• High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient ClinicsStatus unknownNCT04981210
• Effect of Enzyme Replacement Therapy in Patients With Juvenile-onset Pompe DiseaseStatus unknownNCT04942912
• Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage DiseasesActive, not recruitingNCT04929002
• Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaActive, not recruitingNCT04910776Phase 3
• Musculoskeletal Nociceptive Pain in Participants With Neuromuscular DisordersCompletedNCT04907162
• A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)Active, not recruitingNCT04848779
• Getting Global Rare Disease Insights Through Technology StudyStatus unknownNCT04758130Not applicable
• Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.Status unknownNCT04755751
• Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe DiseaseCompletedNCT04724213Not applicable
• Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa TreatmenCompletedNCT04676373Phase 4
• Clinical Specimen Collection From Pompe Disease PatientsStatus unknownNCT04476550
• Gene Transfer Study in Patients With Late Onset Pompe DiseaseActive, not recruitingNCT04174105Phase 1/2
• Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe DiseaseCompletedNCT04120168
• Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe DiseaseWithdrawnNCT04094948Phase 2
• A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)Active, not recruitingNCT04093349Phase 1/2
• ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDActive, not recruitingNCT03911505Phase 3
• Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe DiseaseCompletedNCT03893240Not applicable
• Developing a Management Approach for Patients With "Late-Onset" Pompe DiseaseActive, not recruitingNCT03694561
• Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa TreatmentCompletedNCT03687333Phase 4
• Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
• AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe DiseaseCompletedNCT03533673Phase 1
• Pompe Gene Therapy- Screening for EligibilityCompletedNCT03285126
• Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)CompletedNCT03255213Not applicable
• Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe DiseaseCompletedNCT03147664
• Search for Serum/Plasma Biomarkers in Pompe's DiseaseStatus unknownNCT03045042
• Pompe Telemedicine Developmental StudyCompletedNCT02950298
• Feasability and Interest of Screening for Infantile Pompe's Diseases at BirthCompletedNCT02904395
• Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French GuianaCompletedNCT02903654
• VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe DiseaseTerminatedNCT02898753Phase 1/2
• Investigating Pompe Prevalence in Neuromuscular Medicine Academic PracticesCompletedNCT02838368
• Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)CompletedNCT02824068
• Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)CompletedNCT02801539Not applicable
• Identification of Tongue Involvement in Late-Onset Pompe DiseaseCompletedNCT02765828

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