Trial to Evaluate the Efficacy and Safety of KM-819 Treatment to Slow the Progression of Multiple System Atrophy (MSA)

NCT05695378 | Terminated Phase 2 DMC

• 1 other identifier: KMCP-819-K102
• Study Type: interventional
• Target: 68
• Locations: 1 country
• Sites: 1

Brief Summary

This trial will the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA.

Conditions

Multiple System Atrophy

Keywords

neurodegenerative disorder; Fas-associated factor 1 (FAF1); autonomic dysfunction; KM-819

Outcome Measures

Primary Outcomes (1)

• Percentage change from baseline in putaminal [18F]FP-CIT (18F-FP-CIT Positron Emission Tomography for Correlating Motor and Cognitive Symptoms of Parkinson's Disease) binding

  To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA. The putaminal \[18F\]FP-CIT binding will allow quantification of MSA progression during 36 weeks.

  From Baseline to Week 36

Secondary Outcomes (13)

• Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) scores (UMSARS I + II)

  From Baseline to Week 36

• Change from baseline in the UMSARS I scores

  From Baseline to Week 36

• Change from baseline in the UMSARS II scores

  From Baseline to Week 36

• Percentage change from baseline in putaminal glucose metabolism

  From Baseline to Week 36

• Percentage change from baseline in cerebellar glucose metabolism

  From Baseline to Week 36

Study Arms (3)

Main Study: KM-819

EXPERIMENTAL

Subjects will receive 400 mg of KM-819 orally from Week 0 to Week 36.

Drug: KM-819

Main Study: Placebo

PLACEBO COMPARATOR

Subjects will receive visually identical placebo pills of KM-819 orally.

Drug: Placebo

Ancillary Study: KM-819

EXPERIMENTAL

Subjects will receive 400 mg of KM-819 orally from Week 40 to Week 76.

Drug: KM-819

Interventions

KM-819 DRUG

Subjects will receive KM-819 400 mg orally daily.

Ancillary Study: KM-819; Main Study: KM-819

Placebo DRUG

Subjects will receive Placebo orally daily.

Main Study: Placebo

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must be diagnosed as probable or possible MSA, according to the second consensus criteria for diagnosis of MSA
• Patients who are able to visit the clinic during the study period to be in the study.
• ≥ 30 years and ≤ 80 years of age at the time of signing the Informed Consent
• Antiparkinsonian medications should be stable for, at least, one month before enrollment.
• Body Mass Index (BMI) range of 18.5 to 30 kg/m\^2 inclusive at Screening
• Patient agrees to use acceptable contraceptive methods during the study
• For women, menopause, sterilization confirmed.
• For childbearing women, older than 40, and agreed with more than 2 methods of contraception below and agreed with no desire to be pregnant during and after the study, and, agreed with maintaining medically acceptable methods of contraception during for 90 days after the study.
• Cognitive ability for possible to make self-decision, understand and follow the instruction, to make written signature on consent form.
• If no ability to walk, patients must be accompanied by caregiver by wheelchair on schedule.

You may not qualify if:

• A diagnosis of drug induced parkinsonism by typical neuroleptic treatment or haloperidol medication.
• Women who are pregnant or lactating
• History of suicide attempt. Any recent suicidal ideation (a level of 4 or 5) within the last 3 months prior to Day 1, or has a positive response ('Yes') to either question 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) at check-in (Day 1), or who is at significant risk to commit suicide, as judged by the Investigator using the C-SSRS at Screening.
• Febrile illness or symptomatic viral, bacterial (including upper respiratory infection) or fungal (non-cutaneous) infection.
• Any clinically significant abnormality following the Investigator's review of the physical examination and protocol-defined clinical laboratory tests at Screening or site check-in.
• Patient has a mean pulse rate \< 40
• Patient has a mean corrected QT interval using Fridericia's formula (QTcF) of \> 430 msec (for males) and \> 450 msec (for females).
• History of unexplained syncope, cardiac arrest, unexplained cardiac arrhythmias or torsade de pointes, structural heart disease or a family history of Long QT Syndrome.
• Positive serology test for hepatitis B surface antigen (HBsAg), anti-hepatitis A virus (HAV), immunoglobulin M (IgM), anti-hepatitis C virus (HCV) or anti-human immunodeficiency virus (HIV).
• Known or suspected hypersensitivity to KM-819, or any components of the formulation(s) used.
• Patient has a serious medical or surgical condition.
• Patients unable to understand the consent form, and determined by investigator with too serious problems for participating in the study.
• Patients unable to visit the clinical site on schedule due to the no ability mobilize.
• Patients who had brain surgery history.

Sponsors & Collaborators

• Kainos Medicine Inc.: lead
• Parexel: collaborator

Study Sites (1)

CHA Bundang Medical Center, CHA University

Seongnam-si, Gyeonggi-do, 13497, South Korea

Location

MeSH Terms

Conditions

Multiple System Atrophy; Neurodegenerative Diseases; Primary Dysautonomias

Interventions

KM-819

Condition Hierarchy (Ancestors)

Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies

Study Officials

• Chong Sik Lee

  PI

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2022
• First Posted: January 25, 2023
• Study Start: February 27, 2023
• Primary Completion: August 19, 2024
• Study Completion: August 19, 2024
• Last Updated: September 19, 2024

Record last verified: 2024-09

Locations

KR (1)