Clinical trials for Glycogen storage disease due to acid maltase deficiency
25 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGN/ANCT07478172Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseUniversity of Missouri-Columbia
- RECRUITINGPHASE1, PHASE2NCT07282847A 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)AskBio Inc
- RECRUITINGPhase 2NCT07123155Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)Shionogi
- RECRUITINGPhase 4NCT06666413China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDGenzyme, a Sanofi Company · PI: Trial Transparency email recommended (Toll free for US & Canada)
- RECRUITINGNCT06795152Rare Glycogen Storage Diseases Natural History StudyDuke University · PI: Priya Kishnani, M.D.
- RECRUITINGPHASE1, PHASE2NCT06391736Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy DrugGeneCradle Inc · PI: Guang Yang
- RECRUITINGNCT06121011A Global Prospective Observational Registry of Patients With Pompe DiseaseAmicus Therapeutics
- ACTIVE NOT RECRUITINGEarly Phase 1NCT06109948Study to Assess the Safety, Tolerability, PK and PD of ABX1100Aro Biotherapeutics
- ACTIVE NOT RECRUITINGNCT05943678Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.Medical University of Vienna
- RECRUITINGPhase 3NCT04808505A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18Amicus Therapeutics
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT05793307Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy DrugGeneCradle Inc
- ACTIVE NOT RECRUITINGPhase 4NCT05164055Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)Genzyme, a Sanofi Company · PI: Clinical Sciences & Operations
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT05095727A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)ModernaTX, Inc.
- ACTIVE NOT RECRUITINGNCT04929002Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage DiseasesRigshospitalet, Denmark
- ACTIVE NOT RECRUITINGPhase 3NCT04910776Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaSanofi · PI: Clinical Sciences & Operations
- ACTIVE NOT RECRUITINGNCT04848779A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)Sanofi · PI: Clinical Sciences & Operations
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT04093349A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)Spark Therapeutics, Inc. · PI: Tahseen Mozaffar, MD
- ACTIVE NOT RECRUITINGPhase 3NCT03911505ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDAmicus Therapeutics
- RECRUITINGNCT03564561Natural History of Pompe DiseaseAssistance Publique - Hôpitaux de Paris · PI: Helge Amthor, MD, PhD
- ACTIVE NOT RECRUITINGNCT03694561Developing a Management Approach for Patients With "Late-Onset" Pompe DiseaseDuke University · PI: Priya Kishnani, MD
- ACTIVE NOT RECRUITINGPhase 2NCT03019406A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase AlfaGenzyme, a Sanofi Company · PI: Clinical Sciences & Operations
- RECRUITINGNCT04639336Cognitive and Neurological Pathologies in Pompe DiseaseDuke University · PI: Priya Kishnani, MD
- RECRUITINGNCT01665326Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseDuke University · PI: Priya S Kishnani, MD
- RECRUITINGNCT02399748A Long-term Study for the Outcome of Pompe DiseaseNational Taiwan University Hospital · PI: Yin-Hsiu Chien
- RECRUITINGN/ANCT00231400Pompe Disease Registry ProtocolGenzyme, a Sanofi Company · PI: Study Director