Glycogen Storage Disease clinical trials
The registry lists 36 studies for Glycogen Storage Disease across more than 10 countries. 13 are recruiting or about to start. 16 of them are mainly about Glycogen Storage Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-02. Counting its narrower condition Glycogen Storage Disease Type II too: 187 studies, 40 open.
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Who runs them
Duke University (3) · Institut de Myologie, France (3) · Shionogi (3) · University of Florida (3) · Rigshospitalet, Denmark (2) · CENTOGENE GmbH Rostock (1) · Parasail, LLC (1) · IRCCS Eugenio Medea (1)
What is being tested here
Drugs under test in Glycogen Storage Disease studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| S-606001 | 3 | 0 | 3 |
| Triheptanoin | 0 | 0 | 1 |
| Valproic Acid | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 5
- Nov 2026A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a) · ModernaTX, Inc.
- Dec 2026Neurocognitive Function and Neuroimaging Features in Patients With Glycogen Storage Diseases · Peking Union Medical College Hospital
- Jan 2027Effects of the Carbohydrate Counting Method on Blood Glucose Regulation, Quality of Life, Urinary Glc4 and Other Biomarkers in Children With GSD1 · Şükrü Arman AKSOY
- Jun 2027Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation · IRCCS Azienda Ospedaliero-Universitaria di Bologna
- Aug 2027Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) · Shionogi
Who these studies take
Of the 9 Glycogen Storage Disease studies recruiting now, 4 (44%) set no upper age limit and 4 (44%) stop at 75 or younger. 6 (67%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (22%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (56%) | 5 (56%) |
| 18 | 9 (100%) | 9 (100%) |
| 30 | 8 (89%) | 8 (89%) |
| 40 | 8 (89%) | 8 (89%) |
| 50 | 7 (78%) | 7 (78%) |
| 65 | 7 (78%) | 7 (78%) |
| 75 | 5 (56%) | 5 (56%) |
| 85 | 5 (56%) | 5 (56%) |
Across all 36 studies, recruiting or not: 42% stop at 75 or younger and 31% at 65; 61% take children; 3% are for women only and 0% for men only; 19% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Newly registered2
Studies
Recruiting first, then most recently updated. Showing 36 of 36. search them.
- Neurocognitive Function and Neuroimaging Features in Patients With Glycogen Storage DiseasesRecruitingNCT079082121 country
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- Effects of the Carbohydrate Counting Method on Blood Glucose Regulation, Quality of Life, Urinary Glc4 and Other Biomarkers in Children With GSD1Not yet recruitingNCT078750231 country
- An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)Not yet recruitingNCT07750990Phase 2
- 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 1/21 country
- ATR 1072 in Participants With PRKAG2 SyndromeRecruitingNCT07738107Phase 1/21 country
- A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)Not yet recruitingNCT07787650Phase 1
- 'Glycogen Storage Diseases (GSDs) in Indian Children- Establishing an Indian GSD (I-GSD) Registry'RecruitingNCT063965461 country
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)RecruitingNCT07123155Phase 29 countries
- Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance TechniquesRecruitingNCT073363941 country
- Rare Glycogen Storage Diseases Natural History StudyRecruitingNCT067951521 country
- Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac TransplantationRecruitingNCT068134431 country
- Intravenous VAL-1221 Lafora Expanded Access ProtocolAvailableNCT05930223
- Continuous Glucose Monitoring Alerts, Accuracy, and Patient Outcomes in Adults With Inherited Metabolic DisordersCompletedNCT07614139Not applicable1 country
- Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular DisordersCompletedNCT05199246Not applicable1 country
- Assessment of Safety and Acute Effects of a Knee-hip Powered Soft Exoskeleton in Patients With Neuromuscular DisordersCompletedNCT05200702Not applicable1 country
- Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic DisordersCompletedNCT043996941 country
- A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)Active, not recruitingNCT05095727Phase 1/26 countries
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage DiseasesActive, not recruitingNCT049290021 country
- Pompe Lactation Sub-RegistryCompletedNCT005668781 country
- Glycosade v UCCS in the Dietary Management of Hepatic GSDCompletedNCT02318966Not applicable4 countries
- Biomarker for Glycogen Storage Diseases (BioGlycogen)WithdrawnNCT023851623 countries
- Natural History and Functional Status Study of Patients With Lafora DiseaseCompletedNCT038765223 countries
- McArdle Disease Treatment by Ketogenic DietCompletedNCT04292938Not applicable1 country
- Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)CompletedNCT03255213Not applicable1 country
- VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe DiseaseTerminatedNCT02898753Phase 1/22 countries
- The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)CompletedNCT02432768Phase 21 country
- Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14Status unknownNCT03404856Phase 1/21 country
- Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe DiseaseCompletedNCT00976352Phase 1/21 country
- Sodium Valproate for GSDVCompletedNCT03112889Phase 22 countries
- Search for Serum/Plasma Biomarkers in Pompe's DiseaseStatus unknownNCT030450421 country
- Study of Glycogen Storage Disease Expression in CarriersCompletedNCT020577311 country
- Clinical Evaluation of a Non-Invasive Hypoglycemia Detector in a Glycogen Storage Disease PopulationTerminatedNCT023388171 country
- Prospective Follow-up of Patients With Glycogen Storage Disease Type IIIStatus unknownNCT01563705Not applicable1 country
- Study of Glycogen Storage Disease and Associated DisordersCompletedNCT000013421 country