Status:
Recruiting
Clinicaltrials.gov identifier:
Sponsor:
Neuraly, Inc.
Enrollment:
240
Study Design:
This is a Phase 2 randomized, placebo-controlled double-blind study comparing three parallel assigned arms: NLY01 2.5mg injection, NLY01 5mg injection, and placebo saline injection. Subjects will be randomized 1:1:1 to these three arms. The drug or placebo is delivered via weekly subcutaneous injection.
The study includes individuals aged 30 to 80 with a diagnosis of Parkinson’s disease within 5 years prior to screening. Additionally, participants complete a DaTScan which must be consistent with a PD diagnosis. Notably, individuals who have used dopaminergic treatment or MAO-B inhibitors for more than 28 days, or who have had previous surgical treatment, are excluded from participation in the study. Those found to have major depression or suicidal ideation within 1 year of screening will be excluded.
Rationale:
In addition to the primary outcomes of safety and tolerability, the study is assessing efficacy as measured by change from baseline to 36 weeks in the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III combined score, for each dose of NYL01 compared to placebo. MDS-UPDRS Part II assesses motor aspects of daily living, and Part III assesses motor signs of Parkinson’s.
Secondary efficacy endpoints include change from baseline in: UPDRS Part I: Non-Motor Aspects of Experiences of Daily Living; Clinical Global Impression Scale–Severity (CGI-S); Patient Global Impression of Severity (PGI-S); Schwab and England Activities of Daily Living Scale (SE-ADL); Parkinson’s Disease Questionnaire–39 (PDQ-39); Montreal Cognitive Assessment (MoCA); Scales for Outcomes of Parkinson’s Disease Cognition (SCOPA-COG); Non-Motor Symptoms Scale (NMSS). Other Secondary Assessments include change from baseline in: DaTscan (quantitative); Incidence of anti-NLY01 antibodies; NLY01 population PK assessment. Safety and Tolerability Assessments include: Incidence, severity, and duration of all reported treatment-emergent adverse events (TEAEs); Vital signs, physical examinations, and ECGs; Clinical laboratory assessments; Columbia-Suicide Severity Rating Scale (C-SSRS); Epworth Sleepiness Scale (ESS); Questionnaire for Impulsive-Compulsive Disorders in Parkinson’s Disease–Rating Scale (QUIP-RS); Beck Depression Inventory–II (BDI-II).
Comments:
The unique quality of NYL01 as a pegylated version of the previously-studied exenatide confers a potential advantage over the more well-known compound. By increasing blood-brain-barrier penetration with pegylation, NYL01 may have increased target engagement of central GLP-1 receptors, which in turn could prove more efficacious than its un-pegylated counterpart. This would be an additional benefit to the anticipated well-tolerated safety profile, given the robust safety data for existing GLP-1 agonists.
