Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Recruiting study of Pompe Disease, sponsored by Amicus Therapeutics.
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. According to ClinicalTrials.gov, the official US trial registry.
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
Primary Outcome
Data collection that describe the frequency of AEs/SAEs occurring in Pompe disease patients
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 |
What NCT06121011 shows while recruiting
NCT06121011 is an observational study that tracks outcomes without assigning an intervention. The registered 500 participants enrollment target is mid-sized for trials with a published cap, above the 197-participant average among 14 other Pompe Disease trials with a reported enrollment target (154% higher).
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 names 20 study sites across 13 states, led by 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.
Learn More About Clinical Trials
Similar trials for Pompe Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06121011's enrollment target sits among peer trials
500 2nd of 14 the highest of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
RECRUITING · Phase 2
-
Cognitive and Neurological Pathologies in Pompe Disease
RECRUITING
-
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
RECRUITING
-
Pompe Disease Registry Protocol
RECRUITING
-
Avalglucosidase Alfa Pregnancy Study
RECRUITING
-
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
ACTIVE NOT RECRUITING · Phase 1
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00001230 · 500 participants
Host Response to Infection and Treatment in Filarial Diseases
- NCT00001529 · 500 participants
Improved Methods of Cell Selection for Bone Marrow Transplant Alternatives
- NCT00697411 · 500 participants
Study of Selected X-Linked Disorders: Aicardi Syndrome
- NCT00903110 · 500 participants
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI