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A Global Prospective Observational Registry of Patients With Pompe Disease
NCT06121011 · View on ClinicalTrials.gov ↗
Study Summary
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
Conditions Studied
Interventions
- BIOLOGICAL Cipaglucosidase alfa
- DRUG Miglustat
- BIOLOGICAL Alglucosidase alfa or Avalglucosidase alfa
- OTHER Untreated
Study Locations (20)
Other
- Medizinische Universitaet Wien — Vienna
- Laboratory for Muscle Diseases and Neuropathies — Leuven
- Aarhus Universitets hospital — Aarhus C
- Ruhr-Universität Bochum im St. Josef-Hospital — Bochum
- SphinCS, Institute of Clinical Science in LSD — Höchheim
Pennsylvania
- University of Pennsylvania Perelman Center for Advanced Medicine — Philadelphia
- University of Pittsburgh — Pittsburgh
- UPMC Children's Hospital of Pittsburgh — Pittsburgh
Ohio
- University of Cincinnati Medical Center — Cincinnati
- Cincinnati Children's Hospital — Cincinnati
Arkansas
- University of Arkansas Medical Science — Little Rock
California
- University of California Irvine — Irvine
Florida
- Wolfson Children's Hospital — Jacksonville
Georgia
- Emory University — Atlanta
Indiana
- Indiana University, IU Health Physicians Neurology — Indianapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 500 participants |
| Start Date | 2024-02-16 |
| Est. Completion | 2034-12-20 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06121011
The ClinicalTrials.gov registry entry for NCT06121011 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 500 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Amicus Therapeutics, which has 14 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 4 interventions — of which Cipaglucosidase alfa is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06121011 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Other, Pennsylvania, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06121011 about?
NCT06121011 is a clinical study titled "A Global Prospective Observational Registry of Patients With Pompe Disease". 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...
What is the current status of trial NCT06121011?
This trial is currently recruiting. The enrollment target is 500 participants. The study started on 2024-02-16. Estimated completion is 2034-12-20.
What conditions does trial NCT06121011 study?
This clinical trial studies the following conditions: Pompe Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06121011?
The interventions under investigation include: Cipaglucosidase alfa (BIOLOGICAL), Miglustat (DRUG), Alglucosidase alfa or Avalglucosidase alfa (BIOLOGICAL), Untreated (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06121011?
This trial is sponsored by Amicus Therapeutics, which has 14 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06121011 being conducted?
This trial has 20 study locations across Arkansas, California, Florida, Georgia, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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