Less Frequent Enzyme Dosing in Late-onset Pompe Disease
Tests less frequent alglucosidase alfa dosing in older adults with Pompe disease.
Plain-language title generated from the registry record
Registry title: Treatment Frequency Reduction in Pompe Disease
Key facts
- Where
- Rotterdam, NetherlandsSee the site
- Phase
- Phase 4
Where in Netherlands
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)Rotterdam, NetherlandsTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Erasmus Medical Center
- Trial code
- NCT06575829EU 2024-514255-15-01
- Record updated
- 28 August 2024
- Original description
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 patients with late-onset Pompe disease.Read full original descriptionShow less
Open Label, Single-center Pilot Study to Investigate Alglucosidase Alfa (20 mg/kg) Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease (TRIPOD-Study)
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 patients with late-onset Pompe disease.
Inclusion: LOPD (confirmed diagnosis: enzyme deficiency in any tissue source and/or 2 confirmed disease-causing variants in the GAA gene); Age ≥50 years; Current treatment with alglucosidase alfa at a standard dose of 20 mg/kg once every 2 weeks for ≥4 years; Relatively stable clinical condition over the past year; Able to walk ≥150 m within 6 minutes (6MWT); (Forced) vital capacity (FVC) in sitting position: >55% of expected value and in supine position: >45% of expected value; Willing and able to adhere to the study procedures.
Exclusion: Requiring respiratory support (non-invasive/invasive ventilation) or being at high risk to require respiratory support (ventilation) due to further deterioration of current pulmonary function. Using continuous positive airway pressure (CPAP) support only for obstructive sleep apnea syndrome (OSAS) is permitted; Rapidly progressive muscle weakness; Severely limited muscle strength almost requiring/requiring daily wheelchair use; Comorbidities which are expected to influence the primary outcome measures within the next 2 years.