A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Public ClinicalTrials.gov record NCT03729362. 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.
Official title
A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.
Study identification
- NCT ID
- NCT03729362
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 125 participants
Conditions and interventions
Conditions
Interventions
- Cipaglucosidase Alfa Biological
- Miglustat Drug
- Alglucosidase Alfa Biological
- Placebo Drug
Biological · Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- 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
- Dec 4, 2018 (Actual)
- Primary completion
- Dec 15, 2020 (Actual)
- Completion
- Jan 15, 2021 (Actual)
- Last update posted
- Sep 11, 2025
2018 – 2021
United States locations
- U.S. sites
- 25
- U.S. states
- 19
- U.S. cities
- 24
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Neuromuscular Research Center | Phoenix | Arizona | 85028 | — |
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | — |
| University of California, Irvine | Irvine | California | 92868 | — |
| UF Helath: University of Florida Clinical Research Center | Gainesville | Florida | 32610 | — |
| University of South Florida Research Center | Tampa | Florida | 33612 | — |
| Emory Clinic | Atlanta | Georgia | 30322 | — |
| Indiana University Health Neuroscience Center | Indianapolis | Indiana | 46202 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66205 | — |
| University of Minnesota Clinical Research Unit | Minneapolis | Minnesota | 55455 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| Billings Clinic | Billings | Montana | 59101 | — |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| The Feinstein Institute for Medical Research | Manhasset | New York | 11030 | — |
| NYU School of Medicine | New York | New York | 10017 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| University of Cincinnati Neurology | Cincinnati | Ohio | 45219 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| Penn State Health Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | — |
| University of Texas Health Science Center San Antonio | San Antonio | Texas | 78229 | — |
| University of Utah, Center for Clinical and Translational Sciences | Salt Lake City | Utah | 84108 | — |
| Lysosomal and Rare Disorders Research | Fairfax | Virginia | 22030 | — |
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 48 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03729362, 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 Sep 11, 2025 · Synced Oct 31, 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 public registry record, listed eligibility criteria, and available contact information for NCT03729362 live on ClinicalTrials.gov.