A Global Prospective Observational Registry of Patients With Pompe Disease

Status: Recruiting
Location: See all (40) locations...
Intervention Type: Other, Biological, Drug
Study Type: Observational
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
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping

Locations
United States
Arkansas
University of Arkansas Medical Science
RECRUITING
Little Rock
California
University of California Irvine
RECRUITING
Irvine
Florida
Wolfson Children's Hospital
RECRUITING
Jacksonville
Georgia
Emory University
RECRUITING
Atlanta
Indiana
Indiana University, IU Health Physicians Neurology
RECRUITING
Indianapolis
Missouri
Washington University School of Medicine
RECRUITING
St Louis
North Carolina
Duke University Medical Center
RECRUITING
Durham
New York
NYU Neurogenetics, NYU Langone Medical Center
RECRUITING
New York
Ohio
Cincinnati Children's Hospital
RECRUITING
Cincinnati
University of Cincinnati Medical Center
RECRUITING
Cincinnati
Pennsylvania
University of Pennsylvania Perelman Center for Advanced Medicine
RECRUITING
Philadelphia
University of Pittsburgh
RECRUITING
Pittsburgh
UPMC Children's Hospital of Pittsburgh
RECRUITING
Pittsburgh
Utah
University of Utah
NOT_YET_RECRUITING
Salt Lake City
Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITING
Fairfax
Other Locations
Austria
Medizinische Universitaet Wien
NOT_YET_RECRUITING
Vienna
Belgium
Laboratory for Muscle Diseases and Neuropathies
RECRUITING
Leuven
Denmark
Aarhus Universitets hospital
RECRUITING
Aarhus C
Germany
Ruhr-Universität Bochum im St. Josef-Hospital
RECRUITING
Bochum
SphinCS, Institute of Clinical Science in LSD
RECRUITING
Höchheim
Universitätsklinikum Gießen und Marburg GmhH
RECRUITING
Marburg
Universitat Munchen - Friedrich Baur Institut
NOT_YET_RECRUITING
München
Universitaetsklinikum Ulm
NOT_YET_RECRUITING
Ulm
Greece
Eginition Hospital
NOT_YET_RECRUITING
Athens
Hungary
University of Pécs
NOT_YET_RECRUITING
Pécs
University of Szeged, Szent-Györgyi Albert Clinical Center
NOT_YET_RECRUITING
Szeged
Italy
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
NOT_YET_RECRUITING
Bari
Centre of Expertise for muscular diseases and peripheral neuropathies European Reference Network for Rare Neuromuscular Diseases
NOT_YET_RECRUITING
Napoli
Department of Neurosciences Rita Levi Montalcini, University of Torino
NOT_YET_RECRUITING
Torino
Regional Coordinating Centre for Rare Diseases, university Hospital of Udine, Udine, Italy
NOT_YET_RECRUITING
Udine
Netherlands
Dept of Pediatrics Erasmus MC - Sophia Children's Hospital
NOT_YET_RECRUITING
Rotterdam
Poland
entrum Medyczne Medyk
NOT_YET_RECRUITING
Rzeszów
Slovenia
University Medical Centre Ljubljana, Institute of Clinical Neurophysiology
NOT_YET_RECRUITING
Ljubljana
United Kingdom
Queen Elizabeth Hospital Birmingham
RECRUITING
Birmingham
Cambridge University - Addenbrooke's Hospital
RECRUITING
Cambridge
University Hospital of Wale, Cardiff
NOT_YET_RECRUITING
Cardiff
Great Ormond Street Hospital NHS Foundation Trust
RECRUITING
London
National Hospital for Neurology and Neurosurgery
RECRUITING
London
Royal Free Hospital NHS Foundation Trust
RECRUITING
Manchester
Salford Royal NHS Foundation Trust
RECRUITING
Salford
Contact Information
Primary
For Site
patientadvocacy@amicusrx.com
609-662-2000
Backup
For Patient
patientadvocacy@amicusrx.com
609-662-2000
Time Frame
Start Date: 2024-02-16
Estimated Completion Date: 2034-12-20
Participants
Target number of participants: 500
Treatments
Cipaglucosidase alfa/Miglustat-treated patients
Other Enyzme Replacement Therapy (ERT)-treated patients
Untreated patients (those who are not currently receiving any medical therapy for Pompe disease)
Related Therapeutic Areas
Sponsors
Leads: Amicus Therapeutics

This content was sourced from clinicaltrials.gov