Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Pompe Disease: early-phase trial registry
6 of 15 indexed Pompe Disease trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,140 of 28,707 conditions by registered trial count.
15 US clinical trials · 6 currently recruiting · #1,140 of 28,707 conditions by registered trial count
Pompe Disease: 15 US clinical trials tracked, 6 recruiting.
Pompe Disease is the subject of 15 registered US clinical trials on ClinicalTrials.gov, 6 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 6 in Phase 1-2 (earlier-stage). The most active sponsor is Genzyme, a Sanofi Company, running 5 of these trials.
Enrollment posture brief
Registry condition pompe-disease
OPEN 6 | RECR 40% | LATE 1 | EARLY 6 | RANK #1140 | SPON 5
Volume-matched condition peers
Nearest open-share peer: Achondroplasia (40% recruiting · 10 trials)
Pompe Disease lists 6 open slots (40% of 15 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Pompe Disease on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Pompe Disease is recruiting below its size-band peers.
6 of 15 indexed trials (40%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Pompe Disease research.
Genzyme, a Sanofi Company accounts for 5 of 15 trials (33.3%). 8 distinct sponsors appear on at least one study for this condition label.
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Most Pompe Disease trials plan to enrol fewer than 100 people.
Median target enrolment is 25 among the 15 trials that report a genuine target (sentinel values excluded). 11 of those (73.3%) plan for fewer than 100 participants.
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Pompe Disease splits between interventional and observational designs.
7 interventional and 8 observational studies make up this index (46.7% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
15 total, page 1 of 1
Pompe Disease Registry Protocol
Genzyme, a Sanofi Company
NCT00231400
A Global Prospective Observational Registry of Patients With Pompe Disease
Amicus Therapeutics
NCT06121011
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Duke University
NCT01665326
Avalglucosidase Alfa Pregnancy Study
Sanofi
NCT05734521
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Shionogi
NCT07123155
Cognitive and Neurological Pathologies in Pompe Disease
Duke University
NCT04639336
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Duke University
NCT03694561
AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
AskBio
NCT03533673
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Spark Therapeutics
NCT04093349
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Genzyme, a Sanofi Company
NCT00113035
Drug-drug Interaction Study
Amicus Therapeutics
NCT01380743
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Genzyme, a Sanofi Company
NCT01898364
Growth and Development Study of Alglucosidase Alfa
Genzyme, a Sanofi Company
NCT00486889
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
University of Florida
NCT00976352
Pompe Lactation Sub-Registry
Genzyme, a Sanofi Company
NCT00566878
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 2 |
| Phase 4 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Pompe Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Pompe Disease trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Pompe Disease page
15 registered trials, 6 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 6 Pompe Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Genzyme, a Sanofi Company sponsors the most Pompe Disease trials on record, review their full trial history before evaluating a specific study. See Genzyme, a Sanofi Company's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Pompe Disease condition card totals 15 registered trials; most-linked intervention alglucosidase alfa; top US site state North Carolina. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.