A Global Prospective Observational Registry of Patients With Pompe Disease
Public ClinicalTrials.gov record NCT06121011. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients * To evaluate the long-term real-world effectiveness of Pompe disease treatments * To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs) * To describe the natural history of untreated Pompe disease
Study identification
- NCT ID
- NCT06121011
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 500 participants
Conditions and interventions
Conditions
Interventions
- Cipaglucosidase alfa Biological
- Miglustat Drug
- Alglucosidase alfa or Avalglucosidase alfa Biological
- Untreated Other
Biological · Drug · Other
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 15, 2024
- Primary completion
- Dec 19, 2034
- Completion
- Dec 19, 2034
- Last update posted
- Mar 9, 2026
2024 – 2034
United States locations
- U.S. sites
- 15
- U.S. states
- 12
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Arkansas Medical Science | Little Rock | Arkansas | 72205 | Recruiting |
| University of California Irvine | Irvine | California | 92697 | Recruiting |
| Wolfson Children's Hospital | Jacksonville | Florida | 32207 | Recruiting |
| Emory University | Atlanta | Georgia | 30322 | Recruiting |
| Indiana University, IU Health Physicians Neurology | Indianapolis | Indiana | 46202 | Recruiting |
| Washington University School of Medicine | St Louis | Missouri | 63110 | Recruiting |
| NYU Neurogenetics, NYU Langone Medical Center | New York | New York | 10017 | Recruiting |
| Duke University Medical Center | Durham | North Carolina | 27710 | Recruiting |
| University of Cincinnati Medical Center | Cincinnati | Ohio | 45219 | Recruiting |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | Recruiting |
| University of Pennsylvania Perelman Center for Advanced Medicine | Philadelphia | Pennsylvania | 19104 | Recruiting |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15219 | Recruiting |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | Recruiting |
| University of Utah | Salt Lake City | Utah | 84108 | Recruiting |
| Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax | Virginia | 22030 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 26 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06121011, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 9, 2026 · Synced Sep 5, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06121011 live on ClinicalTrials.gov.