NCT06575829Not yet recruitingPhase 4
Treatment Frequency Reduction in Pompe Disease
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)
Source: ClinicalTrials.gov · Last updated 28 Aug 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 22 Aug 2024
- Start date
- 1 Oct 2024
- Primary completion date
- 31 Dec 2026
- Lead sponsor
- Erasmus Medical Center
- Diseases
- Pompe disease
- NCT number
- NCT06575829
- EU CT number
- 2024-514255-15-01
What you commit to
- Study type: Interventional · Phase 4
- Planned participants: 10
- From 1 Oct 2024 to the primary completion date, 31 Dec 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study design.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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.
Other
- 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
Who the study is looking for
- 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
Age in the registry: 50 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) Coordinator of this site? Confirm its status | Rotterdam | Netherlands | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.