A Phase 2 Study of ONO-2808 in Patients With Multiple System Atrophy
- Conditions
- Multiple System Atrophy (MSA)
- Interventions
- Drug: Placebo
- Registration Number
- NCT05923866
- Lead Sponsor
- Ono Pharmaceutical Co. Ltd
- Brief Summary
This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of multiple doses of ONO-2808 in patients with MSA. This is the first study of ONO-2808 in patients with MSA.
- Detailed Description
The purpose of the study is to evaluate 3 doses of ONO-2808 compared to placebo in MSA patients, including: 1) safety and tolerability, 2) pharmacokinetics, and 3) changes in clinical outcome assessments (COA) and biomarkers considered to be related to the pharmacodynamics and potential efficacy of ONO-2808.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 92
-
Female or male patients with a diagnosis of clinically-established or clinically-probable MSA according to the novel Movement Disorder Society (MDS) criteria for MSA diagnosis (2022), including patients with MSA of either subtype (MSA-P or MSA-C).
-
Patients at the early stages of the disease, defined as a maximum of 5 years since the onset of one of the following symptoms associated with MSA:
- Parkinsonism
- Ataxia
- Orthostatic hypotension and/or urinary dysfunction
-
Patients with an Unified Multiple System Atrophy Rating Scale (UMSARS) 1 total score (excluding item 1.11 sexual function) of ≤ 17.
-
Patients with an anticipated survival of at least 3 years in the opinion of the Investigator.
-
Patients who are able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps and then to turn around and walk at least another 10 steps. Use of assistive devices (e.g., walker or cane) is allowed.
-
Ability to swallow oral medication and be willing to adhere to the study intervention regimen.
- Pregnant or lactating females.
- Patients with a clinically-significant or unstable medical or surgical condition other than MSA that, in the opinion of the Investigator, might preclude safe completion of the study or might affect the results of the study (e.g., pulmonary, cardiovascular [including bradyarrhythmia], macular edema, and significant renal or hepatic dysfunction).
- Neurological diseases/disorders other than MSA, such as Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, normal pressure hydrocephalus, pharmacological, or post-encephalitic parkinsonism.
- Patients with documented liver diseases or cirrhosis.
- Positive results at Screening for active viral infections that include positive human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) and hepatitis B core antibody, and hepatitis C virus (HCV).
- Patients with suicide ideation according to the Investigator's clinical judgment per the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening or who have made a suicide attempt in the 6 months before Screening.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ONO-2808 Arm ONO-2808 - Placebo Arm Placebo -
- Primary Outcome Measures
Name Time Method Vital signs (blood pressure) From screening up to follow-up (Week 28) Summaries of clinically significant and non-clinically significant abnormalities will be provided by each time point.
Vital signs (pulse rate) From screening up to follow-up (Week 28) Summaries of clinically significant and non-clinically significant abnormalities will be provided by each time point.
Vital signs (temperature) From screening up to follow-up (Week 28) Summaries of clinically significant and non-clinically significant abnormalities will be provided by each time point.
Incidence and severity of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs) From screening up to follow-up (Week 28) Incidence of TEAEs, drug-related TEAEs, TEAEs resulting in study treatment discontinuation, TESAEs, and drug-related TESAEs will be tabulated by system organ class (SOC), preferred term (PT), and severity.
Vital signs (respiratory rate) From screening up to follow-up (Week 28) Summaries of clinically significant and non-clinically significant abnormalities will be provided by each time point.
12-lead electrocardiograms (ECGs); parameters such as, but not limited to, heart rate, RR, PR, QRS, QT, and corrected QT intervals (QTcF) From screening up to follow-up (Week 28) The number of patients with normal, abnormal not clinically significant and abnormal clinically significant of ECG results will be tabulated at each time point.
Clinically-significant abnormal physical examination findings From screening up to follow-up (Week 28) The number of patients with normal, abnormal not clinically significant and abnormal clinically significant of physical examination results will be tabulated at each time point.
Clinical laboratory abnormalities (hematology, clinical chemistry, and urinalysis) From screening up to follow-up (Week 28) The number of patients with abnormal laboratory results at any time during the study will be tabulated.
Clinically-abnormal findings in the Columbia Suicide Severity Rating Scale (C-SSRS) From screening up to follow-up (Week 28) Responses to the suicidality assessment scale (C-SSRS) will be listed.
- Secondary Outcome Measures
Name Time Method Plasma concentration of ONO-2808 Week 2, Week 8, Week 12, and Week 24 Descriptive summary statistics will be calculated for ONO-2808 plasma concentrations, by dose level and time point.
ONO-2808 concentration in cerebrospinal fluid (CSF) Week 24 Descriptive summary statistics will be calculated for ONO-2808 CSF concentrations, by dose level and time point.
Related Research Topics
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Trial Locations
- Locations (35)
The Parkinson's Movement and Disorder Institute
🇺🇸Fountain Valley, California, United States
University of Southern California
🇺🇸Los Angeles, California, United States
David Geffen School of Medicine at UCLA
🇺🇸Los Angeles, California, United States
Stanford University School of Medicine
🇺🇸Palo Alto, California, United States
CenExel Rocky Mountain Clinical Research
🇺🇸Englewood, Colorado, United States
Yale School of Medicine - Yale Church Street Research Unit (CRSU)
🇺🇸New Haven, Connecticut, United States
Parkinson's Disease and Movement Disorders Center of Boca Raton
🇺🇸Boca Raton, Florida, United States
Norman Fixel Institute for Neurological Diseases - University of Florida
🇺🇸Gainesville, Florida, United States
Mayo Clinic in Florida
🇺🇸Jacksonville, Florida, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
University of Kansas Medical Center Research Institute
🇺🇸Kansas City, Kansas, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
University of Michigan School of Medicine
🇺🇸Ann Arbor, Michigan, United States
Quest Research Institute
🇺🇸Farmington Hills, Michigan, United States
Mayo Clinic - Rochester
🇺🇸Rochester, Minnesota, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
NYU Langone Health - NYU Dysautonomia Center
🇺🇸New York, New York, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Columbia University
🇺🇸New York, New York, United States
University of Cincinnati College of Medicine
🇺🇸Cincinnati, Ohio, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Ohio State University
🇺🇸Columbus, Ohio, United States
Penn State University - Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
The University of Pennsylvania - Pennsylvania Hospital
🇺🇸Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Virginia Commonwealth University Medical Center
🇺🇸Richmond, Virginia, United States
Evergreen Health
🇺🇸Kirkland, Washington, United States
Swedish Neuroscience Institute, Movement Disorders Clinic
🇺🇸Seattle, Washington, United States
National Hospital Organization Utano National Hospital
🇯🇵Kyoto, Japan
Fujita Health University Hospital
🇯🇵Toyoake, Aichi, Japan
National Hospital Organization Sendai Nishitaga Hospital
🇯🇵Sendai, Miyagi, Japan
Tokyo Metropolitan Neurological Hospital
🇯🇵Fuchū, Tokyo, Japan
National Hospital Organization Osaka Toneyama Medical Center
🇯🇵Toyonaka, Osaka, Japan