A Global Prospective Observational Registry of Patients With Pompe Disease

Status Recruiting

Clinical Trial 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

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06121011
Study type	Observational [Patient Registry]
Source	Amicus Therapeutics
Contact	For Site
Phone	215-921-7600
Email	PompeSiteInfo@amicusrx.com
Status	Recruiting
Phase
Start date	February 16, 2024
Completion date	December 20, 2034

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT01942590` - Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy	Phase 1/Phase 2
Not yet recruiting	`NCT01409486` - Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry	N/A
Completed	`NCT00976352` - Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease	Phase 1/Phase 2
Recruiting	`NCT01665326` - Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Completed	`NCT01758354` - Newborn Screening Assay of Pompe's Disease	N/A
Recruiting	`NCT00231400` - Pompe Disease Registry Protocol
Recruiting	`NCT04476550` - Clinical Specimen Collection From Pompe Disease Patients
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Completed	`NCT01380743` - Drug-drug Interaction Study	Phase 2
Recruiting	`NCT05734521` - Avalglucosidase Alfa Pregnancy Study
Completed	`NCT02742298` - Pompe Disease QMUS and EIM	N/A
Terminated	`NCT02185651` - A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction	Phase 1
Completed	`NCT02654886` - Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.	N/A
Completed	`NCT02405598` - Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease	Phase 4
Completed	`NCT00701129` - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease	Phase 4
Completed	`NCT01451879` - Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies	N/A
Completed	`NCT02240407` - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease	Phase 1
Completed	`NCT05073783` - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Active, not recruiting	`NCT04093349` - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)	Phase 1/Phase 2
Completed	`NCT02363153` - Diet and Exercise in Pompe Disease	N/A