Ignite Creation Date:
2024-05-06 @ 7:45 PM
Last Modification Date:
2024-10-26 @ 3:13 PM
Study NCT ID:
NCT06121011
Status:
RECRUITING
Last Update Posted:
2024-06-26
First Post:
2023-11-02
Brief Title:
A Global Prospective Observational Registry of Patients With Pompe Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
A Global Prospective Observational Registry of Patients With Pompe Disease
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 AEsserious 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
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 AEsserious 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None