POM-005
Research type
Research Study
Full title
A global prospective observational registry of patients with Pompe disease
IRAS ID
332410
Contact name
Mark Roberts
Contact email
Sponsor organisation
Amicus Therapeutics, Inc.
Duration of Study in the UK
10 years, 0 months, 22 days
Research summary
This is a global, multicenter, prospective, observational registry of patients with Pompe disease.
The study population in this registry will consist of patients with a diagnosis of Pompe disease (late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD)).
The registry will be conducted at approximately 100 sites that manage patients with Pompe disease, including patients with either LOPD or IOPD. The goal of this registry is to assess clinical outcomes in patients with Pompe disease, including patients with late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD), regardless of current or previous therapy. No mandatory visits, tests, or assessments are required for this registry.
The use of real-world data for this registry will enable the evaluation of the long-term safety and effectiveness of Pompe disease treatments in a broad population of patients with Pompe disease.
The prospective design enables the capture of important characteristics of patients with Pompe disease ([LOPD] or [IOPD]).
The registry enrollment target will be approximately 500 consecutive patients diagnosed with LOPD or IOPD. Patients will be followed prospectively for at least 5 years from the date of signed informed consent (enrollment) or until the withdrawal of consent, enrollment in an interventional clinical study with an investigational product for Pompe disease, loss to follow-up, death, or the end of the registry, whichever comes first.
This registry is planned to start in Q4 2023, with last patient enrolled Q4 2028, end of data collection in Q1 2033, and final report of registry results Q4 2033.REC name
London - Riverside Research Ethics Committee
REC reference
23/PR/1287
Date of REC Opinion
22 Dec 2023
REC opinion
Further Information Favourable Opinion