Glycogen Storage Disease Type II clinical trials
The registry lists 162 studies for Glycogen Storage Disease Type II across more than 10 countries. 34 are recruiting or about to start. 142 of them are mainly about Glycogen Storage Disease Type II (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-13.
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· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
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Status
Who runs them
Genzyme, a Sanofi Company (37) · Duke University (13) · Amicus Therapeutics (12) · National Taiwan University Hospital (9) · University of Florida (8) · LMU Klinikum (5) · Sanofi (4) · BioMarin Pharmaceutical (4)
What is being tested here
Drugs under test in Glycogen Storage Disease Type II 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 |
| Avalglucosidase alfa | 1 | 1 | 7 |
| Faricimab | 1 | 0 | 1 |
| Alglucosidase alfa | 0 | 4 | 21 |
| Cipaglucosidase alfa | 0 | 3 | 4 |
| Albuterol | 0 | 0 | 3 |
| Miglustat | 0 | 1 | 2 |
| Ranibizumab | 0 | 0 | 2 |
| Clenbuterol | 0 | 0 | 2 |
| Reveglucosidase alfa | 0 | 0 | 2 |
Drug classes tested here Angiogenesis inhibitors 1 open · Bronchodilators 5
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.
The 10 nearest, phase 3 first
- Mar 2027ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD · Phase 3 · Amicus Therapeutics
- Jul 2027A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 · Phase 3 · Amicus Therapeutics
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug · GeneCradle Inc
- Dec 2026A Long-term Study for the Outcome of Pompe Disease · National Taiwan University Hospital
- Dec 2026Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases · Assistance Publique Hopitaux De Marseille
- Dec 2026Treatment Frequency Reduction in Pompe Disease · Erasmus Medical Center
- May 2027The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. · LMU Klinikum
- Jun 2027A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease · Astellas Gene Therapies
- Jun 2027Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) · Genzyme, a Sanofi Company
Who these studies take
Of the 23 Glycogen Storage Disease Type II studies recruiting now, 16 (70%) set no upper age limit and 6 (26%) stop at 75 or younger. 15 (65%) take children under 18. 2 (9%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 14 (61%) | 12 (52%) |
| 18 | 19 (83%) | 17 (74%) |
| 30 | 18 (78%) | 16 (70%) |
| 40 | 18 (78%) | 16 (70%) |
| 50 | 19 (83%) | 17 (74%) |
| 65 | 19 (83%) | 17 (74%) |
| 75 | 18 (78%) | 16 (70%) |
| 85 | 16 (70%) | 14 (61%) |
Across all 162 studies, recruiting or not: 30% stop at 75 or younger and 26% at 65; 62% take children; 4% are for women only and 0% for men only; 12% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Glycogen Storage Disease Type II studies take
Of 29 observational studies under Glycogen Storage Disease Type II, half reached primary completion within 108 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Interventional, no phase | 34 mo20–42 · 16 studies | — | — |
| Observational | 108 mo33–… · 29 studies | — | — |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 14, 2026.
Newly registered1
- MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)Oct 23, 2026NCT07892651
Studies
Recruiting first, then most recently updated. Showing 50 of 162. Browse all 162 → or search them.
- Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy DrugRecruitingNCT06391736Phase 1/21 country
- Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle DiseasesRecruitingNCT06833489Not applicable1 country
- Pompe Disease Registry ProtocolRecruitingNCT0023140044 countries
- Expanded Access for ATB200/AT2221 for the Treatment of IOPDAvailableNCT043279733 countries
- Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseAvailableNCT03865836
- MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)Not yet recruitingNCT078926511 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
- Pompe Disease RWE Long-term Monitoring ProgramRecruitingNCT078343201 country
- A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)Not yet recruitingNCT07787650Phase 1
- A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe DiseaseRecruitingNCT07354724Phase 11 country
- Phrenic Nerve and Diaphragm Electrophysiology in Pompe DiseaseRecruitingNCT076649301 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
- Pompe Pregnancy Sub-RegistryRecruitingNCT005670739 countries
- Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe DiseaseRecruitingNCT016653261 country
- China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPDRecruitingNCT06666413Phase 41 country
- A Global Prospective Observational Registry of Patients With Pompe DiseaseRecruitingNCT0612101112 countries
- Cognitive and Neurological Pathologies in Pompe DiseaseRecruitingNCT046393361 country
- A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18RecruitingNCT04808505Phase 36 countries
- Avalglucosidase Alfa Pregnancy StudyRecruitingNCT057345211 country
- Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular DisordersEnrolling by invitationNCT066056121 country
- The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Enrolling by invitationNCT07072676Not applicable1 country
- Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment IntervalsRecruitingNCT06875245Phase 41 country
- A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMDRecruitingNCT06557369Not applicable1 country
- FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesRecruitingNCT065391691 country
- Treatment Frequency Reduction in Pompe DiseaseNot yet recruitingNCT06575829Phase 4
- Natural History of Pompe DiseaseRecruitingNCT035645611 country
- Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)Not yet recruitingNCT06178432Early Phase 11 country
- Nutritional Therapy in Late-onset Pompe DiseaseNot yet recruitingNCT06130228Phase 2
- Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe DiseaseRecruitingNCT05567627Not applicable1 country
- Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseaseRecruitingNCT052729691 country
- Inspiratory Muscle Training (IMT) in Adult People With Pompe DiseaseRecruitingNCT05951790Not applicable1 country
- Higher Dose of Alglucosidase Alpha for Pompe DiseaseNot yet recruitingNCT05017402
- A Long-term Study for the Outcome of Pompe DiseaseRecruitingNCT023997481 country
- A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)CompletedNCT048487799 countries
- Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy DrugActive, not recruitingNCT05793307Phase 1/21 country
- Gene Transfer Study in Patients With Late Onset Pompe DiseaseActive, not recruitingNCT04174105Phase 1/22 countries
- A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe DiseaseActive, not recruitingNCT06150820Not applicable11 countries
- Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseActive, not recruitingNCT07478172Not applicable1 country
- 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 36 countries
- Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)Active, not recruitingNCT05164055Phase 41 country
- Developing a Management Approach for Patients With "Late-Onset" Pompe DiseaseActive, not recruitingNCT036945611 country
- LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONSCompletedNCT076853141 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 YearsActive, not recruitingNCT07652814Phase 41 country
- Study to Assess the Safety, Tolerability, PK and PD of ABX1100CompletedNCT06109948Early Phase 12 countries
- AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe DiseaseCompletedNCT03533673Phase 11 country
- Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaActive, not recruitingNCT04910776Phase 39 countries
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)CompletedNCT04138277Phase 323 countries
- A 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 AlfaActive, not recruitingNCT03019406Phase 25 countries