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Treatment frequency Reduction In POmpe disease (TRIPO-Study)
EU CTIS ID: 2024-514255-15-01
What this study is testing
The primary objective is to explore whether less frequent treatment with ERT (once every 4 weeks instead of once every 2 weeks) does not lead to increased progression of muscle strength, muscle function and pulmonary function in a selected group of elderly patients with LOPD who is in a stable condition (not requiring wheelchair use and respiratory support during the day while awake and seated), and who is at no immediate risk of losing the ability to carry out important activities of daily living (ADLs). If this less frequent treatment regimen (once every 4 weeks instead of once every 2 weeks) proves to be safe for a period of 9 months, and does not lead to increased disease progression, we aim to investigate whether treatment can eventually be discontinued.
- Therapeutic use (Phase IV)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- To be eligible for this study, a subject must meet all of the following criteria: • 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 ERT at a standard dose of 20 mg/kg once every 2 weeks for ≥4 years. Patients who switched to a different type of ERT during these 4 years must have received the 'new' ERT for at least 1 year and had a stable clinical course after the switch • Relatively stable clinical condition over the past year • Able to walk ≥150 m within 6 minutes (6MWT) • Non-invasive ventilation in supine position or, in case of no (non-invasive) ventilation,(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
You likely can't join if
- • Rapidly progressive muscle weakness. • Severely limited muscle strength almost requiring/requiring daily wheelchair use. • Requiring respiratory support (non-invasive/invasive ventilation) during the day while awake and seated, or being at high risk to require respiratory support (ventilation) either during the day or at night due to further deterioration of current pulmonary function. • Comorbidities which are expected to influence the primary outcome measures within the next 2 years.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.