Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Pompe Disease (Late-onset): full-pipeline trial registry
2 of 9 indexed Pompe Disease (Late-onset) trials are currently recruiting.
Corpus placement: #1,916 of 28,707 conditions by registered trial count.
9 US clinical trials · 2 currently recruiting · #1,916 of 28,707 conditions by registered trial count
Pompe Disease (Late-onset): 9 US clinical trials tracked, 2 recruiting.
Pompe Disease (Late-onset) is the subject of 9 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 3 are in Phase 3-4 (later-stage) and 3 in Phase 1-2 (earlier-stage). The most active sponsor is Genzyme, a Sanofi Company, running 3 of these trials.
Enrollment posture brief
Registry condition pompe-disease-late-onset
OPEN 2 | RECR 22% | LATE 3 | EARLY 3 | RANK #1916 | SPON 3
Volume-matched condition peers
Nearest open-share peer: Acne (22% recruiting · 27 trials)
Pompe Disease (Late-onset) lists 2 open slots (22% of 9 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 (Late-onset) 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 (Late-onset) is recruiting below its size-band peers.
2 of 9 indexed trials (22.2%) 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 (Late-onset) research.
Genzyme, a Sanofi Company accounts for 3 of 9 trials (33.3%). 6 distinct sponsors appear on at least one study for this condition label.
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Most Pompe Disease (Late-onset) trials plan to enrol fewer than 100 people.
Median target enrolment is 20 among the 9 trials that report a genuine target (sentinel values excluded). 8 of those (88.9%) plan for fewer than 100 participants.
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Pompe Disease (Late-onset) research is mostly interventional.
7 interventional and 2 observational studies make up this index (77.8% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
9 total, page 1 of 1
Pompe Pregnancy Sub-Registry
Genzyme, a Sanofi Company
NCT00567073
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
NCT07282847
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Astellas Gene Therapies
NCT06150820
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Amicus Therapeutics
NCT03911505
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Duke University
NCT03694561
Gene Transfer Study in Patients With Late Onset Pompe Disease
Astellas Gene Therapies
NCT04174105
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Spark Therapeutics
NCT04093349
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Genzyme, a Sanofi Company
NCT00158600
Late-Onset Treatment Study Extension Protocol
Genzyme, a Sanofi Company
NCT00455195
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 3 | 2 |
| Phase 4 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Pompe Disease (Late-onset) records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Pompe Disease (Late-onset) 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 (Late-onset) page
9 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 Pompe Disease (Late-onset) trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Genzyme, a Sanofi Company sponsors the most Pompe Disease (Late-onset) 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 (Late-onset) condition card totals 9 registered trials; most-linked intervention alglucosidase alfa; top US site state Pennsylvania. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.