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NCT06121011 · ClinicalTrials.gov registry record
A Global Prospective Observational Registry of Patients With Pompe Disease
A clinical trial of Pompe Disease, sponsored by Amicus Therapeutics.
- Recruiting
- Registry status
- 500
- Enrollment target
- 20
- Study locations
NCT06121011 is a study of Pompe Disease that is actively recruiting participants, run by Amicus Therapeutics. The registered enrollment target is 500 participants, above the 197-participant average among 14 other Pompe Disease trials with a reported enrollment target (154% higher). The trial reports 20 study locations across 13 states.
The verdict
NCT06121011, a study of Pompe Disease, is actively recruiting participants, sponsored by Amicus Therapeutics.
- RECRUITING
- Registry status
- 500 participants
- Enrollment target
- 20
- Study locations
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 |
Interested in This Trial?
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, categorized as an unspecified phase. The registered enrollment target is 500 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 197-participant average among 14 other Pompe Disease trials with a reported enrollment target (154% higher). The listed sponsor is Amicus Therapeutics, which has 34 total studies on file at ClinicalTrials.gov.
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.
NCT06121011 reports 20 study locations spanning 13 distinct geographic areas - top geographies include Other, Pennsylvania, Ohio.
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.
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).
Who is sponsoring clinical trial NCT06121011?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
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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Read our methodology - how this data is sourced, computed, and verified.
Related
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