A randomized, Phase 2a, double-blinded, biomarker-driven, placebo-controlled study to assess safety, CNS penetration, and target engagement of Mirivadelgat in Parkinson’s disease – SLEIPNIR-2, a sub-protocol in the SLEIPNIR platform trial

Parkinson's Disease

Incidence and severity of adverse events (AEs) and serious adverse events (SAEs); treatment adherence based on dosing compliance and study drug discontinuation rate., CSF-to-plasma concentration ratio of the Mirivadelgat metabolite AD-835 at Week 12, measured by LC-MS., Change from baseline to Week 12 in CSF total 4-HNE protein adduct levels, measured by validated ELISA assay.

Change from baseline to Week 12 in CSF levels of 4-HNE, measured by ELISA, or other relevant method., Change from baseline to Week 12 in CSF levels of TBARS (nmol malondialdehyde [MDA] equivalents per mL), measured using a validated thiobarbituric acid reactive substances (TBARS) spectrophotometric assay., Change from baseline to Week 12 in CSF levels of 4-HNE-α-synuclein using a custom sandwich ELISA assay., Change from baseline to Week 12 in the following brain bioenergetic indices: • Inorganic phosphate to ATP ratio (Pi/ATP), reflecting ATP turnover and energy consumption. • Phosphocreatine to inorganic phosphate (PCr/Pi) ratio, reflecting cellular energetic state and phosphorylation potential. • Phosphocreatine to ATP ratio (PCr/ATP), reflecting ATP resynthesis reserve and energetic state. measured by phosphorus magnetic resonance spectroscopy (31P-MRS) in the posterior brain.

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