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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

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30 recruiting trials

for Glycogen Storage Diseases

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford NCT01793168n/a1 site📍 Sioux Falls Sanford Health Accuracy of Lactate Meter in GSDIa NCT06843330n/a1 site📍 Hartford Connecticut Children's Medical Center · Tests: blood glucose meter; blood lactate meter Danon Disease Natural History Study NCT06214507n/a16 sites📍 Phoenix📍 La Jolla📍 Aurora Rocket Pharmaceuticals Inc. · Tests: No intervention 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) NCT07282847Phase 1, Phase 29 sites📍 Phoenix📍 Irvine📍 Palo Alto AskBio Inc · Tests: AB-1009 (GAA Gene) Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease NCT07459582n/a1 site📍 Hartford Connecticut Children's Medical Center · Tests: Lactate Meter, Accu Chek GBE Deficiency (GSD IV and APBD) Natural History Study NCT02683512n/a1 site📍 Durham Duke University · Tests: No intervention ATR 1072 in Participants With PRKAG2 Syndrome NCT07738107Phase 1, Phase 21 site📍 Kansas City Atrium Therapeutics · Tests: ATR 1072 Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I NCT06852612NA2 sites📍 Bern📍 Zurich Insel Gruppe AG, University Hospital Bern · Tests: Fructose, Fructose and galactose Pompe Disease Registry Protocol NCT00231400n/a282 sites📍 Birmingham📍 Phoenix📍 Phoenix Genzyme, a Sanofi Company Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment Intervals NCT06875245Phase 41 site📍 Prague Faculty Hospital Kralovske Vinohrady · Tests: Faricimab Injection A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease NCT07354724Phase 14 sites📍 Irvine📍 St Louis📍 Durham Denali Therapeutics Inc. · Tests: DNL952 A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD NCT06557369NA1 site📍 Bergamo Oculox Technologies SA · Tests: reSEES Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease NCT01665326n/a1 site📍 Durham Duke University · Tests: Observational Pompe Pregnancy Sub-Registry NCT00567073n/a35 sites📍 Phoenix📍 Jacksonville📍 Atlanta Genzyme, a Sanofi Company A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease NCT06092034Phase 26 sites📍 La Jolla📍 Boston📍 Philadelphia Rocket Pharmaceuticals Inc. · Tests: RP-A501 Cognitive and Neurological Pathologies in Pompe Disease NCT04639336n/a1 site📍 Durham Duke University Brain Metabolism Observed at 3 Tesla or 7 Tesla in Health and Metabolic Disease NCT05085704n/a2 sites📍 New York📍 Dallas Weill Medical College of Cornell University · Tests: Magnetic resonance imaging French Observatory for Patients with Type 3 Glycogenosis NCT06616545n/a3 sites📍 Clamart📍 Le Kremlin-Bicêtre📍 Paris Institut de Myologie, France Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program NCT06636383n/a19 sites📍 Orange📍 Denver📍 Hartford Ultragenyx Pharmaceutical Inc · Tests: No Intervention Use of Continuous Glucose Monitoring to Evaluate Postprandial Response to Raw Cornstarch Supplementation in Adult Glycogen Storage Disease Type I NCT07645898n/a1 site📍 Naples Federico II University Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology NCT07136844NA1 site📍 Liège Centre Hospitalier Universitaire de Liege · Tests: Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT), The 10-meter test (10MWT), The 4 sta… A Long-term Study for the Outcome of Pompe Disease NCT02399748n/a1 site📍 Taipei National Taiwan University Hospital A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 NCT04808505Phase 314 sites📍 Gainesville📍 Atlanta📍 Durham Amicus Therapeutics · Tests: Cipaglucosidase alfa, Miglustat Avalglucosidase Alfa Pregnancy Study NCT05734521n/a1 site📍 Bridgewater Sanofi · Tests: avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666) Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation NCT06813443n/a3 sites📍 Bologna📍 Roma📍 Florence IRCCS Azienda Ospedaliero-Universitaria di Bologna China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD NCT06666413Phase 41 site📍 Shanghai Genzyme, a Sanofi Company · Tests: Avalglucosidase alfa Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease NCT07664930n/a1 site📍 Pavia IRCCS National Neurological Institute "C. Mondino" Foundati… GSD VI and GSD IX Natural History NCT04454216n/a1 site📍 Durham Duke University · Tests: No intervention Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease NCT07478172NA1 site📍 Columbia University of Missouri-Columbia · Tests: Whole-body Electrical Muscle Stimulation Exercise A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) NCT06735755Phase 1, Phase 23 sites📍 Orange📍 Farmington📍 Houston Beam Therapeutics Inc. · Tests: BEAM-301: Single dose of BEAM-301 administered by IV
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