NCT06121011 A Global Prospective Observational Registry of Patients With Pompe Disease
| NCT ID | NCT06121011 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Amicus Therapeutics |
| Condition | Pompe Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 500 participants |
| Start Date | 2024-02-16 |
| Primary Completion | 2034-12-20 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 500 participants in total. It began in 2024-02-16 with a primary completion date of 2034-12-20.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
Eligibility Criteria
Inclusion Criteria: * Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping Exclusion Criteria: * Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06121011 clinical trial?
This trial is open to participants of all sexes, studying Pompe Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06121011 currently recruiting?
Yes, NCT06121011 is actively recruiting participants. Contact the research team at patientadvocacy@amicusrx.com for enrollment information.
Where is the NCT06121011 trial being conducted?
This trial is being conducted at Little Rock, United States, Irvine, United States, Jacksonville, United States, Atlanta, United States and 11 additional locations.
Who is sponsoring the NCT06121011 clinical trial?
NCT06121011 is sponsored by Amicus Therapeutics. The trial plans to enroll 500 participants.