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A Global Prospective Observational Registry of Patients With Pompe Disease

StatusRecruiting
PhaseNot specified
Started
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Record History

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  1. Study sites: 42 to 41v15
  2. Study sites: 41 to 42v14
  3. Study sites: 40 to 41v13
  4. Study sites: 25 to 40v12
  5. Study sites: 24 to 25v11
Show 9 earlier versionsHide 9 earlier versions
  1. Study sites: 22 to 24v10
  2. Study sites: 19 to 22v9
  3. Study sites: 18 to 19v8
  4. Study sites: 16 to 18v7
  5. Site details revised at 1 of 16 sitesv6
  6. Study sites: 8 to 16v5
  7. Study sites: 7 to 8v4
  8. Study sites: 2 to 7v3
  9. Trial status: Not Yet Recruiting to Recruitingv2 Show 4 more changesShow less
    • Primary and study completion dates: Nov 20, 2033 to Dec 20, 2034
    • Primary endpoint revised
    • Start date changed from estimate of Nov 20, 2023 to Feb 16, 2024
    • Site details revised at 2 of 2 sites
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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
Trial Details
NCT Number NCT06121011
Lead Sponsor Amicus Therapeutics
Conditions
(from sponsor)		 Pompe Disease
Related terms Glycogen Storage Disease Type II
Enrollment 500 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov