A Study to Evaluate Safety and Efficacy of KM-819 in Healthy Adults and Participants with Parkinson's Disease

FAScinate Therapeutics

Status and phase

Active, not recruiting

Phase 2

Conditions

Parkinson Disease

Treatments

Drug: Placebo

Drug: KM-819

Study type

Interventional

Funder types

Industry

Identifiers

NCT05670782

FASCP-819-K103

Details and patient eligibility

About

The goal of this study is to test KM-819 in halting or slowing the progression of Parkinson's disease.

The study evaluates the safety and tolerability of multiple ascending doses of KM-819 in healthy older adults and participants with Parkinson's disease.

Full description

The overall study will consist of three parts (Part 1a, Part 1b and Part 2).

Part 1 of this study will evaluate the safety, tolerability and plasma PK of multiple ascending doses (MAD) of KM-819 in healthy older adults (Part 1a) and participants with Parkinson's disease (Part 1b).

Part 1a is a randomized, double-blind, Multiple Ascending Dose (MAD) study in healthy older adults that will include 3 cohorts.
Part 1b is a randomized, double-blind, MAD study in participants with Parkinson's disease that will include 3 cohorts.

Part 2 of the study is a randomized, double-blind, multiple dose study in participants with Parkinson's disease that will include 2 cohorts. It is designed to test the safety, tolerability, plasma PK and pharmacodynamic effects of KM-819 in participants with Parkinson's disease. The study will also assess the degree to which those treated with KM-819 will experience gains in overall daily function within the context of improved Parkinson's disease motor and non-motor symptoms in comparison to placebo. Participants will be randomized to receive KM-819 or matching placebo at doses to be determined based on the findings from Part 1 in a 2:1 ratio.

Enrollment

314 estimated patients

Sex

All

Ages

40+ years old

Volunteers

Accepts Healthy Volunteers

Inclusion criteria

Participant is a healthy volunteer or has a clinical diagnosis of idiopathic Parkinson's disease.
Participant is on a stable dose of medications to treat Parkinson's disease at least 8 weeks prior to randomization
Presence of idiopathic Parkinson's disease Hoehn and Yahr Stage ≤ 4
History or current use of dopamine/dopaminergic drugs, levodopa with decarboxylase inhibitor or dopaminergic agonists, with a stable dosage for at least 30 days prior to Screening
Body mass index (BMI) within the range 18.5 to 35 kg/m2 (inclusive)
A male participant must not have a pregnant or breastfeeding partner and must agree to use a highly effective contraception method starting from Screening and refrain from donating sperm during this period
A female participant is eligible to participate if she is not pregnant, not breastfeeding

Exclusion criteria

Diagnosis of neurodegenerative disorder other than idiopathic Parkinson's disease resulting in dementia or atypical parkinsonism
Life-time history of a suicide attempt as determined by the Columbia-Suicide Severity Rating Scale (C-SSRS) for the Screening
Evidence of cognitive decline defined by the Montreal Cognitive Assessment (MoCA) score ≤25 for healthy normal population (Part 1a) and ≤21 for the patient population (Part 1b and Part 2)
History of levodopa-induced motor fluctuations or dyskinesia
Prior surgical treatment for Parkinson's disease
Clinically significant brain abnormalities on or contraindication to a structural magnetic resonance imaging (MRI)
Significant respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, pancreatic, musculoskeletal, genitourinary, immunological or dermatological disorders.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Double Blind

314 participants in 8 patient groups

Part 1a: Cohort 1.1a Dose 400 mg

Experimental group

Description:

Healthy older adult participants will receive oral 400 mg dose of KM-819 or matching placebo once-daily for 7 days, after fasting for 2 hours prior to administration of study intervention and will be required to fast for 1 hour after administration of study intervention.

Treatment:

Drug: KM-819

Drug: Placebo

Part 1a: Cohort 1.2a Dose 600 mg

Experimental group

Description:

Healthy older adult participants will receive oral 600 mg dose of KM-819 or matching placebo once-daily for 7 days, after fasting for 2 hours prior to administration of study intervention and will be required to fast for 1 hour after administration of study intervention.

Treatment:

Drug: KM-819

Drug: Placebo

Part 1a: Cohort 1.3a Dose 800 mg

Experimental group

Description:

Healthy older adult participants will receive oral 800 mg dose of KM-819 or matching placebo once-daily for 7 days, after fasting for 2 hours prior to administration of study intervention and will be required to fast for 1 hour after administration of study intervention.

Treatment:

Drug: KM-819

Drug: Placebo

Part 1b: Cohort 1.1b Dose 200 mg

Experimental group

Description:

Participants with Parkinson's disease will receive oral 200 mg dose of KM-819 or matching placebo once-daily for 7 days, after fasting for 2 hours prior to administration of study intervention and will be required to fast for 1 hour after administration of study intervention.

Treatment:

Drug: KM-819

Drug: Placebo

Part 1b: Cohort 1.2b Dose 400 mg

Experimental group

Description:

Participants with Parkinson's disease will receive oral 400 mg dose of KM-819 or matching placebo once-daily for 7 days, after fasting for 2 hours prior to administration of study intervention and will be required to fast for 1 hour after administration of study intervention.

Treatment:

Drug: KM-819

Drug: Placebo

Part 1b: Cohort 1.3b Dose 600 mg

Experimental group

Description:

Participants with Parkinson's disease will receive oral 600 mg dose of KM-819 or matching placebo once-daily for 7 days, after fasting for 2 hours prior to administration of study intervention and will be required to fast for 1 hour after administration of study intervention.

Treatment:

Drug: KM-819

Drug: Placebo

Part 2: Cohort 2.1 Dose X

Experimental group

Description:

Participants with Parkinson's disease will receive oral doses of KM-819 (dose to be determined based on the findings from Part 1) or matching placebo once-daily for 730 days and will be allowed to take study intervention with or without fasting.

Treatment:

Drug: KM-819

Drug: Placebo

Part 2: Cohort 2.2 Dose Y

Experimental group

Description:

Treatment:

Drug: KM-819

Drug: Placebo