Pompe disease: trials for referral
33 open trials in the registries we read. Each row links to its registry record.
Registry data only. Frontlight does not judge whether a patient can take part: the trial team decides.
Where the open trials are
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33 trials shown
Phase 20 sites
Not yet recruiting
NCT07750990
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Shionogi
Registry updated 13 Oct 2026
Registry record: ClinicalTrials.govPhase 1/29 sites
RecruitingUnited States
NCT07282847
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)
AskBio Inc
Registry updated 13 Oct 2026
Registry record: ClinicalTrials.govPhase 10 sites
Not yet recruiting
NCT07787650
A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)
Shionogi
Registry updated 26 Aug 2026
Registry record: ClinicalTrials.govPhase 16 sites
RecruitingGermany, Netherlands, United States
NCT07354724
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Denali Therapeutics Inc.
Registry updated 17 Aug 2026
Registry record: ClinicalTrials.govPhase 228 sites
RecruitingBelgium, Germany, Denmark, Spain, France, United Kingdom
NCT07123155
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Shionogi
Registry updated 23 Jun 2026
Registry record: ClinicalTrials.govPhase 41 site
RecruitingChina
NCT06666413
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Genzyme, a Sanofi Company
Registry updated 23 Mar 2026
Registry record: ClinicalTrials.govPhase 11 site
RecruitingUnited States
NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
University of California, San Francisco
Registry updated 17 Mar 2026
Registry record: ClinicalTrials.govPhase 318 sites
RecruitingGermany, Denmark, France, United Kingdom, Italy, Netherlands
NCT04808505
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Amicus Therapeutics
Registry updated 22 Jan 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingBelgium
NCT07136844
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
Centre Hospitalier Universitaire de Liege
Registry updated 22 Aug 2025
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlyGermany
NCT07072676
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
LMU Klinikum
Registry updated 18 Jul 2025
Registry record: ClinicalTrials.govPhase 1/21 site
RecruitingChina
NCT06391736
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
GeneCradle Inc
Registry updated 3 Jul 2025
Registry record: ClinicalTrials.govPhase 41 site
Not yet recruitingNetherlands
NCT06575829
Treatment Frequency Reduction in Pompe Disease
Erasmus Medical Center
Registry updated 28 Aug 2024
Registry record: ClinicalTrials.govEarly Phase 11 site
Not yet recruitingChina
NCT06178432
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
Huashan Hospital
Registry updated 21 Dec 2023
Registry record: ClinicalTrials.govNo phase282 sites
RecruitingUnited Arab Emirates, Argentina, Australia, Belgium, Bulgaria, Brazil
NCT00231400
Pompe Disease Registry Protocol
Genzyme, a Sanofi Company
Registry updated 28 Oct 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingFrance
NCT06833489
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Assistance Publique Hopitaux De Marseille
Registry updated 28 Oct 2026
Registry record: ClinicalTrials.govNo phase1 site
Not yet recruitingJordan
NCT07892651
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Axiom Bioregistry
Registry updated 23 Oct 2026
Registry record: ClinicalTrials.govPhase 41 site
Not yet recruitingNetherlands
2024-520365-31-01
Pompe disease; new Outcomes measures, Treatment Effect and Novel Targeted Investigations for Advancing Long-term follow-up (POTENTIAL).
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Registry updated 1 Oct 2026
Registry record: EU CTISNo phase1 site
RecruitingTaiwan
NCT07834320
Pompe Disease RWE Long-term Monitoring Program
National Taiwan University Hospital
Registry updated 22 Sept 2026
Registry record: ClinicalTrials.govPhase 214 sites
RecruitingBelgium, Germany, Denmark, Spain, France, Italy
2025-522146-40-00
A phase 2, randomized, double-blind study of S-606001 as an add-on to enzyme replacement therapy in patients with late-onset Pompe disease.
Shionogi B.V.
Registry updated 18 Sept 2026
Registry record: EU CTISNo phase1 site
RecruitingItaly
NCT07664930
Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease
IRCCS National Neurological Institute "C. Mondino" Foundation
Registry updated 24 Jun 2026
Registry record: ClinicalTrials.govNo phase35 sites
RecruitingAustralia, Belgium, Brazil, Czechia, Germany, United Kingdom
NCT00567073
Pompe Pregnancy Sub-Registry
Genzyme, a Sanofi Company
Registry updated 16 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT01665326
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Duke University
Registry updated 8 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT05619900
Registry of Patients Diagnosed With Lysosomal Storage Diseases
University of California, San Francisco
Registry updated 8 Apr 2026
Registry record: ClinicalTrials.govNo phase41 sites
RecruitingAustria, Belgium, Germany, Denmark, United Kingdom, Greece
NCT06121011
A Global Prospective Observational Registry of Patients With Pompe Disease
Amicus Therapeutics
Registry updated 10 Mar 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT04639336
Cognitive and Neurological Pathologies in Pompe Disease
Duke University
Registry updated 4 Mar 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT05734521
Avalglucosidase Alfa Pregnancy Study
Sanofi
Registry updated 16 Jan 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT05989620
Long-Term Development of Muscular Dystrophy Outcome Assessments
Virginia Commonwealth University
Registry updated 18 Nov 2025
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlyGermany
NCT06605612
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
LMU Klinikum
Registry updated 10 Sept 2025
Registry record: ClinicalTrials.govPhase 41 site
RecruitingDenmark
2023-508000-37-01
GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
Rigshospitalet
Registry updated 22 Aug 2025
Registry record: EU CTISPhase 41 site
Not yet recruitingNetherlands
2024-518269-92-00
Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Registry updated 16 Dec 2024
Registry record: EU CTIS
The same sites as a list
- United States13 open trials
- Germany9 open trials
- Netherlands8 open trials
- Italy7 open trials
- France6 open trials
- Belgium6 open trials
- Denmark5 open trials
- United Kingdom5 open trials
- China4 open trials
- Taiwan4 open trials
- Spain2 open trials
- Australia2 open trials
- Brazil2 open trials
- Jordan2 open trials
- Hungary2 open trials
- Croatia2 open trials
- Canada1 open trial
- India1 open trial
- South Korea1 open trial
- Thailand1 open trial
- Bulgaria1 open trial
- Argentina1 open trial
- Malaysia1 open trial
- Vietnam1 open trial
- Colombia1 open trial
- Japan1 open trial
- Lebanon1 open trial
- Romania1 open trial
- Czechia1 open trial
- Greece1 open trial
- Austria1 open trial
- Slovenia1 open trial
- Poland1 open trial
Who treats this
Institutions with trials or publications in this disease (OpenAlex, registries). Institutions only, no people.
Cambridge University Hospitals NHS Foundation Trust
Cambridge, United Kingdom
1 trial · 5 publications · ROR
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
6 trials · 4 publications · ROR
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, United States
6 trials · 10 publications · ROR
National Hospital for Neurology and Neurosurgery
London, United Kingdom
2 trials · 3 publications · ROR
What changed in the last 7 days
Registry facts, one per line, newest first.
Closed for recruitment
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
NCT07478172 · No phase · United States
Source: ClinicalTrials.govPhase changed: Phase 1/2 → Phase 1
NCT07354724 · Phase 1 · Germany, Netherlands, United States
Source: ClinicalTrials.govStatus changed: Recruiting → Not yet recruiting
Treatment Frequency Reduction in Pompe Disease
NCT06575829 · Phase 4 · Netherlands
Source: ClinicalTrials.govClosed for recruitment
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT04093349 · Phase 1/2 · Canada, Germany, Denmark, France, United Kingdom, Italy
Source: ClinicalTrials.govStatus changed: Running, not recruiting → Completed
NCT04138277 · Phase 3 · Argentina, Austria, Australia, Belgium, Canada, Germany
Source: ClinicalTrials.govStatus changed: Completed → Running, not recruiting
NCT03911505 · Phase 3 · Australia, Canada, Germany, Italy, Japan, United States
Source: ClinicalTrials.govOpened for recruitment
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT04093349 · Phase 1/2 · Canada, Germany, Denmark, France, United Kingdom, Italy
Source: ClinicalTrials.govOpened for recruitment
Treatment Frequency Reduction in Pompe Disease
NCT06575829 · Phase 4 · Netherlands
Source: ClinicalTrials.gov
Results card
Trials that finished or stopped in the last 3 years, with the reason the registry gives and linked publications.
Phase 1Ended 12 Mar 2026
Early Phase 1Ended 13 Feb 2026
Phase 2Ended 30 Dec 2025
Withdrawn before start0 participants
NCT04094948
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
Reason given: hasn't got the funding
No phaseEnded 2 Jun 2025
No phaseEnded 16 Apr 2025
No phaseEnded 1 Jan 2025
Phase 3Ended 31 Dec 2024
Completed119 participants
NCT04138277
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
- Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials. · Therapeutic advances in rare disease, 2026
- Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease. · Therapeutic advances in rare disease, 2026
No phaseEnded 15 Dec 2024
Completed87 participants
NCT07015970
Real-life Follow-up of the Physical Activity in Type 1 Diabetes Participants Equipped With an Insulin Pump With Hybrid Closed Loop
Phase 1/2Ended 22 Aug 2024
Completed29 participants
NCT02675465
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
- Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials. · Therapeutic advances in rare disease, 2026
Phase 4Ended 25 Jul 2024
Completed41 participants
NCT04676373
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
No phaseEnded 9 Feb 2024
No phaseEnded 31 Jan 2024
No phaseEnded 1 Dec 2023
Completed101 participants
NCT04724213
Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease
Not medical advice. fetched 7 Nov 2026