Phase I, KM-819 in Healthy Subjects for Parkinson's Disease
Condition: Parkinson's Disease
MeSH terms: Parkinson Disease
Study type: Interventional
Phase: Phase 1

Summary: 
      This first in human, single-center, randomized, placebo-controlled, double blind, sequential
      group Phase 1 study in healthy subjects will be conducted to evaluate the safety,
      tolerability, PK, and PD following the escalation of single and multiple doses of KM-819.

      The study will consist of 2 parts. In Part A, up to 5 cohorts of young adult male subjects,
      and 1 single dose cohort of elderly male or post menopausal female subjects will receive
      escalating single doses of KM-819. In Part B, up to 4 cohorts of healthy young adult male
      subjects and 1 multiple dose cohort of elderly male or post menopausal female subjects will
      receive escalating multiple doses of KM-819. Part B will be conducted after completion of all
      cohorts of young adult male subjects in Part A.

      Dose escalation to the next level will be determined using safety, tolerability, and PK data
      of the previous cohort.

      Part A, Single Ascending Dose (SAD) Up to 40 healthy young adult male subjects and 8 healthy
      elderly male or post menopausal female subjects will be enrolled and randomized to receive
      either KM-819 or placebo.

      Each of the 5 dose escalation cohorts consists of 8 healthy young adult male subjects; 6
      subjects will receive 10, 30, 100, 200, or 400 mg of KM-819 and 2 subjects will receive
      placebo. In each single dose cohort, dosing of subjects will be sentinel, i.e., 2 subjects
      will be dosed on the first day (1 subject will receive active treatment and 1 subject will
      receive placebo) and the remaining 6 subjects will be dosed at least 24 hours after the first
      2 subjects.

      Cohorts will be dosed sequentially with escalating doses. Eight elderly male or
      post-menopausal female subjects will be enrolled into an additional cohort; 6 subjects will
      receive 200 mg KM-819 and 2 subjects will receive placebo.

      Part A consists of a Screening period of up to 28 days, and a 3 day Confinement period when
      subjects are hospitalized for study activities. Subjects are required to return for
      outpatient visits on Day 4, 7 and for the Follow up Visit on Day 14.

      Part B, Multiple Ascending Dose (MAD) Up to 32 healthy young adult male subjects and 8
      healthy elderly male or post menopausal female subjects will be enrolled and randomized to
      receive either KM-819 or placebo.

      Each of the 4 dose escalation cohorts consists of 8 healthy young adult male subjects; 6
      subjects will receive 30, 100, 200, or 400 mg of KM-819 once a day (QD) for 7 days and 2
      subjects will receive placebo. Cohorts will be dosed sequentially with escalating doses.

      Eight elderly male or post-menopausal female subjects will be enrolled into an additional
      cohort; 6 subjects will receive 200 mg KM-819 QD for 7 days and 2 subjects will receive
      placebo.
    


Intervention name(s): KM-819; Placebo
Intervention type(s): Drug; Drug
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Basic Science
Keywords: FAF1; Fas (TNFRSF6)-associated factor 1; FAF1 inhibitor; KM-819