Disease: Parkinson's disease
Type of study: Interventional trial
Coordinating investigators: Irene Flønes
Study director: Charalampos Tzoulis
Background: There are currently no disease-modifying therapies (DMTs) capable of halting the progression of Parkinson’s disease (PD), with over 70 negative trials. A fundamental challenge is that investigational compounds advance to efficacy trials without adequate evidence of target engagement (i.e., interaction of the treatment with its intended biological target/pathway) in the patient brain. Without this evidence, the therapeutic potential of a compound remains speculative, rendering costly and time-consuming trials futile. To address this pressing need, we have established SLEIPNIR, a platform trial designed to assess target engagement of tentative DMTs in PD.
SLEIPNIR will test 3-4 candidate DMTs per operational cycle (1.5 years). The chosen compounds are supported by robust preclinical data establishing safety and neuroprotective effects in relevant models and have undergone phase I clinical safety testing. This strategic intervention is poised to significantly accelerate the path to breakthrough treatments for PD.
The primary objective is to evaluate whether the tested compounds engage their intended biological targets in the patient brain.
Primary endpoint: The between-group (active treatment vs. placebo) difference in appropriate measures of target penetration and/or engagement.
Status: The protocol was established in 2024. Recruitment is planned to start in Q3 2025 (depending on funding).
Participating centre
- Haukeland University Hospital, Bergen
Funding
- The Regional Health Authority of Western Norway
- The Research Council of Norway, Neuro-SysMed
- Haukeland University Hospital
- Norwegian Parkinson Association