Clinical trials: Pompe Disease (Late-onset)
Glycogen Storage Disease Type II, Pompe Disease
Pompe Disease Registry Protocol
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both…
- Trial name
- NCT00231400
- Where
- 282 sites in United States, Argentina and 42 more
- Volunteers
- 2,000 people
- Sponsor
- Genzyme, a Sanofi Company
Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.
- Trial name
- NCT00765414
- Testing
- Myozyme
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Genzyme, a Sanofi Company
Glycogen Storage Disease Type II, Pompe Disease (Late-onset)
Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease
The goal of this multicentre, randomized and controlled cross-over trial is to evaluate the efficacy of a programme of Inspiratory Muscle Training in subjects with Late On-set Pompe Disease (LOPD).
- Trial name
- LOPD01 (NCT05951790)
- Testing
- Inspiratory Muscle Training (IMT), Inspiratory Muscle Training (IMT) + Air Stacking
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione Don Carlo Gnocchi ETS
Late-Onset Pompe Disease, Lysosomal Disease
High Dose Inspiratory Muscle Training in LOPD
Study Objectives: 1) assess the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely in Late-onset Pompe Disease (LOPD) and 2) determine its effects on respiratory and patient-reported outcomes.
- Trial name
- NCT05431127
- Testing
- IMT therapy using the Pr02 mobile device
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Duke University
Late-Onset Pompe Disease
LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS
Late-onset Pompe disease (LOPD) is an inherited metabolic disorder caused by deficiency of acid alpha-glucosidase (GAA). In addition to skeletal and respiratory muscle involvement, previous studies suggest that patients with LOPD…
- Trial name
- NCT07685314
- Where
- Spain
- Volunteers
- 477 people
- Sponsor
- Hospitales Universitarios Virgen del Rocío
Pompe Disease (Late-onset), GAA Deficiency
Treatment Frequency Reduction in Pompe Disease
The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of…
- Conditions
- Pompe Disease (Late-onset), GAA Deficiency, Glycogen Storage Disease Type II, Acid Maltase Deficiency
- Trial name
- TRIPOD (NCT06575829)
- Testing
- Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks
- Volunteers
- 10 people
- Sponsor
- Erasmus Medical Center
Pompe Disease (Late-onset)
Pompe Gene Therapy- Screening for Eligibility
The purpose of this study is to determine eligibility for the future clinical trial of gene therapy in adults with late-onset Pompe disease.
- Trial name
- NCT03285126
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Duke University
Pompe Disease, Pompe Disease (Late-onset)
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT).
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II
- Trial name
- NCT04093349
- Testing
- SPK-3006
- Where
- 29 sites in United States, Canada and 6 more
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II)
A Study of rhGAA in Patients With Late-Onset Pompe Disease
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- NCT00250939
- Testing
- Myozyme
- Where
- Netherlands
- Volunteers
- 5 people
- Sponsor
- Genzyme, a Sanofi Company
Pompe Disease (Late-onset), Pompe Disease Late-Onset
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
- Conditions
- Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
- Trial name
- NCT07282847
- Testing
- AB-1009 (GAA Gene)
- Where
- 9 sites in United States
- Volunteers
- 12 people
- Sponsor
- AskBio Inc