Multiple System AtrophyPhase 2NCT05695378
Trial to Evaluate the Efficacy and Safety of KM-819 Treatment to Slow the Progression of Multiple System Atrophy (MSA)
A Phase 2 study testing KM-819 in people with Multiple System Atrophy, run by Kainos Medicine Inc. at 1 site in South Korea.
Trial summary
This trial will the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA. Read more about this study
- Who is it for?
- Ages 30 to 80 years
- Conditions the study is for
- Multiple System Atrophy
- What is being tested?
- KM-819
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 1 siteSouth Korea
- Study dates
- 27 Feb 2023to 19 Aug 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country.
South Korea1 site
CHA Bundang Medical Center, CHA University
Seongnam-si, Gyeonggi-do, 13497
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of KM-819 Treatment to Slow the Progression of Multiple System Atrophy
This trial will the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA.
Read the detailed description
This is a randomized, double-blind, placebo-controlled phase II trial. This trial will be performed in two part: Main study and Ancillary study.
Main Study: Following a 4-week screening period, subjects will be stratified by MSA subtype (MSA-P, -C [MSA-Parkinsonian type, MSA-cerebellar ataxia]) and randomly assigned in a 1:1 ratio either to KM-819 or Placebo groups.
During a treatment period of 36 weeks, subjects will receive pills of either KM-819 or Placebo for oral administration every day from baseline visit. Following this, there will be a safety follow-up period at Week 40.
Ancillary Study: This ancillary study will provide additional information on the continuing efficacy and safety of KM-819. Subjects in either treatment group in the main study who complete the study are eligible to participate in a follow-up, all-subjects-on-treatment (KM-819), open-label ancillary study.
All subjects in the ancillary study will receive KM-819 for additional 36 weeks regardless of their treatment allocation during the main study. During a treatment period of 36 weeks, subjects will receive pills of KM-819 for oral administration every day from visit at Weeks 40.
- Conditions
- Multiple System Atrophy
- Being tested
- KM-819, Placebo
- Keywords
- neurodegenerative disorder, Fas-associated factor 1 (FAF1), autonomic dysfunction, KM-819
- Medical subject headings
- Multiple System Atrophy, Neurodegenerative Diseases, Primary Dysautonomias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KM-819Drug
Subjects will receive KM-819 400 mg orally daily.
PlaceboDrug
Subjects will receive Placebo orally daily.
| Study group | Role | What they receive |
|---|---|---|
| Main Study: KM-819 | Experimental | Subjects will receive 400 mg of KM-819 orally from Week 0 to Week 36. Receives: Drug: KM-819 |
| Main Study: Placebo | Placebo comparator | Subjects will receive visually identical placebo pills of KM-819 orally. Receives: Drug: Placebo |
| Ancillary Study: KM-819 | Experimental | Subjects will receive 400 mg of KM-819 orally from Week 40 to Week 76. Receives: Drug: KM-819 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From Baseline to Week 36
Secondary outcomes (13)
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) scores (UMSARS I + II)
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA. The Unified Multiple System Atrophy Rating Scale (UMSARS) was developed to provide a surrogate measure of disease progression in multiple system atrophy. It is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability (Historical Review), UMSARS-II (14 items) assesses motor impairment based on a clinical examination. Each item scores 0-4. UMSARS-I total score is 48 and UMSARS-II total score is 56. Higher scores on the UMSARS indicate greater disability.
Measured: From Baseline to Week 36
Change from baseline in the UMSARS I scores
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA. The Unified Multiple System Atrophy Rating Scale (UMSARS) was developed to provide a surrogate measure of disease progression in multiple system atrophy. It is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability (Historical Review), UMSARS-II (14 items) assesses motor impairment based on a clinical examination. Each item scores 0-4. UMSARS-I total score is 48. Higher scores on the UMSARS indicate greater disability.
Measured: From Baseline to Week 36
Change from baseline in the UMSARS II scores
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA. The Unified Multiple System Atrophy Rating Scale (UMSARS) was developed to provide a surrogate measure of disease progression in multiple system atrophy. It is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability (Historical Review), UMSARS-II (14 items) assesses motor impairment based on a clinical examination. Each item scores 0-4. UMSARS-II total score is 56. Higher scores on the UMSARS indicate greater disability.
Measured: From Baseline to Week 36
Percentage change from baseline in putaminal glucose metabolism
To evaluate the efficacy of KM-819 compared to placebo for slowing the putaminal of MSA, as measured by [18F]FDG PET (fluorodeoxyglucose (FDG)-positron emission tomography (PET)).
Measured: From Baseline to Week 36
Percentage change from baseline in cerebellar glucose metabolism
To further evaluate the efficacy of KM-819 compared to placebo for slowing the cerebellar progression of MSA, as measured by [18F]FDG PET.
Measured: From Baseline to Week 36
Change from baseline in the Unified Parkinson Disease Rating Scale (UPDRS) III score
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA, measured by clinical scales. The UPDRS is the most widely applied rating instrument for PD. The Total UPDRS III scale includes 18 items. Each item scores 0-4. UPDRS III total score is 72. The highest score refers to the most severe level of disability due to Parkinson's disease.
Measured: From Baseline to Week 36
Change from baseline in the Scale for the Assessment and Rating of Ataxia (SARA) score
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA, measured by clinical scales. The SARA is a tool for assessing ataxia. It has eight categories with an accumulative scores ranging from 0 (no ataxia) to 40 (most severe ataxia). The eight categories were: Gait (0-8 score); Stance (0-6 score); Sitting (0-4 score); Speech disturbance (0-6 score); Finger chase(0-4 score); Nose-finger test (0-4 score); Fast alternating hand movements (0-4 score); Heel-shin slide (0-4 score). Mild dependence: 5.5 or lower, Moderate dependence: 14.25 or lower, Maximal dependence: 23 of higher.
Measured: From Baseline to Week 36
Change from baseline in the Montreal Cognitive Assessment (MoCA) score
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA, measured by clinical scales. MoCA can be used to detect dementia in a clinical setting. A total score of 30 with over 26 is normal. The lower score refers the severe cognitive impairment.
Measured: From Baseline to Week 36
Change from baseline in the Beck's Depression Inventory (BDI-II) score
To evaluate the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA, measured by clinical scales. The BDI-II is scored by summing the ratings for the 21 items. Each item is rated on a 4-point scale ranging from 0 to 3. The maximum total score is 63. Measures of 0-13: Minimal, 14-19: Mild, 20-28: Moderate, 29-63: Severe.
Measured: From Baseline to Week 36
Number of subjects with adverse events (AEs) and serious AEs (SAEs)
To evaluate the safety of KM-819 in subjects with MSA.
Measured: From Screening (Day -4) to Week 40
Maximum observed concentration (Cmax)
To describe the pharmacokinetics parameter (Cmax) of KM-819 using sparse PK sampling.
Measured: From Baseline to Week 36
Area under the concentration-time curve (AUC)
To describe the pharmacokinetics parameter (AUC) of KM-819 using sparse PK sampling.
Measured: From Baseline to Week 36
Time of maximum observed concentration (Tmax)
To describe the pharmacokinetics parameter (Tmax) of KM-819 using sparse PK sampling.
Measured: From Baseline to Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2023
- Study start27 Feb 2023
- Primary completion19 Aug 2024
- Study completion19 Aug 2024
Why it stopped: Sponsor has decided to withdraw the study due to safety issue and re-initiate with a new clinical study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple System AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kainos Medicine Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
- Investigators
- Chong Sik Lee, Principal investigator, PI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05695378
- Sponsor's protocol ID
- KMCP-819-K102
- Status last verified
- Sep 2024
- First submitted
- 22 Dec 2022
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of KM-819 Treatment to Slow the Progression of Multiple System Atrophy
- ClinicalTrials.gov ID
- NCT05695378
- Acronym / sponsor ID
- KMCP-819-K102
- Conditions
- Multiple System Atrophy
- Keywords
- neurodegenerative disorder, Fas-associated factor 1 (FAF1), autonomic dysfunction, KM-819
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor has decided to withdraw the study due to safety issue and re-initiate with a new clinical study
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Kainos Medicine Inc.Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- 27 Feb 2023 (actual)
- Primary completion
- 19 Aug 2024 (actual)
- Study completion
- 19 Aug 2024 (actual)
- First posted
- 25 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial will the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA.
Detailed description
This is a randomized, double-blind, placebo-controlled phase II trial. This trial will be performed in two part: Main study and Ancillary study.
Main Study: Following a 4-week screening period, subjects will be stratified by MSA subtype (MSA-P, -C [MSA-Parkinsonian type, MSA-cerebellar ataxia]) and randomly assigned in a 1:1 ratio either to KM-819 or Placebo groups.
During a treatment period of 36 weeks, subjects will receive pills of either KM-819 or Placebo for oral administration every day from baseline visit. Following this, there will be a safety follow-up period at Week 40.
Ancillary Study: This ancillary study will provide additional information on the continuing efficacy and safety of KM-819. Subjects in either treatment group in the main study who complete the study are eligible to participate in a follow-up, all-subjects-on-treatment (KM-819), open-label ancillary study.
All subjects in the ancillary study will receive KM-819 for additional 36 weeks regardless of their treatment allocation during the main study. During a treatment period of 36 weeks, subjects will receive pills of KM-819 for oral administration every day from visit at Weeks 40.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Main Study: KM-819 | Experimental | Drug: KM-819Subjects will receive 400 mg of KM-819 orally from Week 0 to Week 36. |
| Main Study: Placebo | Placebo comparator | Drug: PlaceboSubjects will receive visually identical placebo pills of KM-819 orally. |
| Ancillary Study: KM-819 | Experimental | Drug: KM-819Subjects will receive 400 mg of KM-819 orally from Week 40 to Week 76. |
Who may qualify
- Ages
- 30 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KM-819Subjects will receive KM-819 400 mg orally daily. | Drug | Ancillary Study: KM-819, Main Study: KM-819 |
| PlaceboSubjects will receive Placebo orally daily. | Drug | Main Study: Placebo |
Primary outcome
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
Time frame: From Baseline to Week 36
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.
13 secondary outcomes
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) scores (UMSARS I + II)
Time frame: From Baseline to Week 36
Change from baseline in the UMSARS I scores
Time frame: From Baseline to Week 36
Change from baseline in the UMSARS II scores
Time frame: From Baseline to Week 36
Percentage change from baseline in putaminal glucose metabolism
Time frame: From Baseline to Week 36
Percentage change from baseline in cerebellar glucose metabolism
Time frame: From Baseline to Week 36
Change from baseline in the Unified Parkinson Disease Rating Scale (UPDRS) III score
Time frame: From Baseline to Week 36
Change from baseline in the Scale for the Assessment and Rating of Ataxia (SARA) score
Time frame: From Baseline to Week 36
Change from baseline in the Montreal Cognitive Assessment (MoCA) score
Time frame: From Baseline to Week 36
Change from baseline in the Beck's Depression Inventory (BDI-II) score
Time frame: From Baseline to Week 36
Number of subjects with adverse events (AEs) and serious AEs (SAEs)
Time frame: From Screening (Day -4) to Week 40
Maximum observed concentration (Cmax)
Time frame: From Baseline to Week 36
Area under the concentration-time curve (AUC)
Time frame: From Baseline to Week 36
Time of maximum observed concentration (Tmax)
Time frame: From Baseline to Week 36
Results and safety
The study was stopped early (reason given: Sponsor has decided to withdraw the study due to safety issue and re-initiate with a new clinical study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chong Sik Lee principal investigator
PI
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing KM-819 in people with Multiple System Atrophy, run by Kainos Medicine Inc. at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor has decided to withdraw the study due to safety issue and re-initiate with a new clinical study). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: From Baseline to Week 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.