Disease: Parkinson's disease
Type of study: Interventional trial
Principal investigator: Simon Kverneng
Study director/Chief investigator: Charalampos Tzoulis
Background: There are currently no approved diseasemodifying therapies (DMTs) for Parkinson’s disease (PD), and all efficacy trials of tentative DMTs to date have yielded negative results. At the same time, the number of candidate investigational medicinal products (IMPs) and early-phase DMT trials is rapidly increasing. Given the high cost and failure rate of efficacy trials in PD, there is an urgent need for a strategic approach to identify and prioritize compounds with the greatest potential to impact disease progression. Two critical factors must be assessed early in clinical
development:
- Target penetration: does the compound and/or its active metabolites reach the human central nervous system (CNS)?
- Target engagement: does the compound engage and modulate its intended biological target in the human CNS, in the context of the disease?
SLEIPNIR is a biomarker-driven, Phase 2a multi-arm platform trial designed to address this translational gap by evaluating CNS penetration and target engagement of investigational products in individuals with PD. By testing multiple IMPs in parallel against a shared placebo group, the platform enables efficient, rigorous, and ethical triaging of candidate compounds prior to advancing to large, costly clinical efficacy trials.
This approach promotes evidence-based decisionmaking, reduces participant burden, and accelerates the development of effective DMTs for PD.
The primary objectives: (I) assess safety and tolerability of IMPs, (ii) evaluate the CNS bioavailability of IMPs with potential DMT-effect in participants with PD, and (iii) assess evidence of target engagement in the CNS.
Primary endpoints: (i) the between-group (active treatment vs. placebo) difference in incidence and severity of adverse events and treatment compliance, (ii) cerebrospinal (CSF)-to-plasma concentration ratio of IMPs and/or their active metabolites at steady state (Week 12) measured by appropriate methodology for each compound, and (iii) change from baseline to Week 12 in biomarkers of target engagement, specific for each compound.
Status: SLEIPNIR will launch with three parallel study arms testing three different IMPs. The master protocol and two sub-protocols were finalized and prepared for CTIS submission during 2025, while the third subprotocol is in finalization. Recruitment is planned to start in Q2 2026, pending CTIS approval.
Participating centre
- Haukeland University Hospital, Bergen, Norway
Funding
- Research Council of Norway
- Western Norway Regional Health Authority
- Norwegian Parkinson’s Association
- Cure Parkinson’s (UK-based charity)
More information
For more information or to join the study, please contact: neurosysmedstudier@helse-bergen.no