A Global Prospective Observational Registry of Patients With Pompe Disease

Status: Recruiting
Location: See all (22) 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
Saint 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
Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITING
Fairfax
Other Locations
Germany
Ruhr-Universität Bochum im St. Josef-Hospital
RECRUITING
Bochum
SphinCS, Institute of Clinical Science in LSD
RECRUITING
Hochheim
Universitätsklinikum Gießen und Marburg GmhH
RECRUITING
Marburg
United Kingdom
Queen Elizabeth Hospital Birmingham
RECRUITING
Birmingham
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
PompeSiteInfo@amicusrx.com
215-921-7600
Backup
For Patient
patientadvocacy@amicusrx.com
215-921-7600
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