Eine Phase-2-Studie zu ONO-2808 bei Patienten mit Multisystematrophie
Eine doppelblinde, placebokontrollierte Parallelgruppenstudie der Phase 2 zur Bewertung der Sicherheit, Verträglichkeit, Pharmakokinetik, Pharmakodynamik und potenziellen Wirksamkeit mehrerer Dosen von ONO-2808 bei Patienten mit multipler Systematrophie (MSA)
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.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Detaillierte Beschreibung
This study will consist of a core part, an extension part, and an additional extension part. The purpose of the core part 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. The purpose of the extension part and the additional extension part is to evaluate 3 doses of ONO-2808 in MSA patients, including: 1) safety and tolerability and 2) changes in COAs and biomarkers considered to be related to the pharmacodynamics and potential efficacy of ONO-2808.
Studientyp
Einschreibung (Tatsächlich)
Phase
- Phase 2
Kontakte und Standorte
Studienorte
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Aichi-ken
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Toyoake, Aichi-ken, Japan
- Fujita Health University Hospital
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Kyoto
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Kyoto, Kyoto, Japan
- National Hospital Organization Utano National Hospital
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Miyagi
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Sendai, Miyagi, Japan
- National Hospital Organization Sendai Nishitaga Hospital
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Osaka
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Toyonaka, Osaka, Japan
- National Hospital Organization Osaka Toneyama Medical Center
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Tokyo
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Fuchū, Tokyo, Japan
- Tokyo Metropolitan Neurological Hospital
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California
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Fountain Valley, California, Vereinigte Staaten, 92708
- The Parkinson's Movement and Disorder Institute
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Los Angeles, California, Vereinigte Staaten, 90033
- University of Southern California
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Los Angeles, California, Vereinigte Staaten, 90095
- David Geffen School of Medicine at UCLA
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Palo Alto, California, Vereinigte Staaten, 94304
- Stanford University School of Medicine
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Colorado
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Englewood, Colorado, Vereinigte Staaten, 80113
- CenExel Rocky Mountain Clinical Research
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Connecticut
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New Haven, Connecticut, Vereinigte Staaten, 06519
- Yale School of Medicine - Yale Church Street Research Unit (CRSU)
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Florida
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Boca Raton, Florida, Vereinigte Staaten, 33486
- Parkinson's Disease and Movement Disorders Center of Boca Raton
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Gainesville, Florida, Vereinigte Staaten, 32608
- Norman Fixel Institute for Neurological Diseases - University of Florida
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Jacksonville, Florida, Vereinigte Staaten, 32224
- Mayo Clinic in Florida
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Miami, Florida, Vereinigte Staaten, 33136
- University of Miami Miller School of Medicine
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Georgia
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Atlanta, Georgia, Vereinigte Staaten, 30329
- Emory University School of Medicine
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Kansas
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Kansas City, Kansas, Vereinigte Staaten, 66160
- University of Kansas Medical Center Research Institute
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Massachusetts
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Boston, Massachusetts, Vereinigte Staaten, 02115
- Brigham and Women's Hospital
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Boston, Massachusetts, Vereinigte Staaten, 02114
- Massachusetts General Hospital
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Michigan
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Ann Arbor, Michigan, Vereinigte Staaten, 48109
- University of Michigan School of Medicine
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Farmington Hills, Michigan, Vereinigte Staaten, 48334
- Quest Research Institute
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Minnesota
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Rochester, Minnesota, Vereinigte Staaten, 55905
- Mayo Clinic - Rochester
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Nebraska
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Omaha, Nebraska, Vereinigte Staaten, 68198
- University of Nebraska Medical Center
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New York
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New York, New York, Vereinigte Staaten, 10032
- Columbia University
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New York, New York, Vereinigte Staaten, 10019
- Icahn School of Medicine at Mount Sinai
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New York, New York, Vereinigte Staaten, 10016
- NYU Langone Health - NYU Dysautonomia Center
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Ohio
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Cincinnati, Ohio, Vereinigte Staaten, 45219
- University of Cincinnati College of Medicine
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Cleveland, Ohio, Vereinigte Staaten, 44195
- Cleveland Clinic
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Columbus, Ohio, Vereinigte Staaten, 43201
- Ohio State University
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Pennsylvania
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Hershey, Pennsylvania, Vereinigte Staaten, 17033
- Penn State University - Milton S. Hershey Medical Center
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Philadelphia, Pennsylvania, Vereinigte Staaten, 19107
- The University of Pennsylvania - Pennsylvania Hospital
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Texas
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Dallas, Texas, Vereinigte Staaten, 75390
- UT Southwestern Medical Center
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Virginia
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Richmond, Virginia, Vereinigte Staaten, 23298
- Virginia Commonwealth University Medical Center
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Washington
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Kirkland, Washington, Vereinigte Staaten, 98034
- Evergreen Health
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Seattle, Washington, Vereinigte Staaten, 98122
- Swedish Neuroscience Institute, Movement Disorders Clinic
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Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
Inclusion Criteria:
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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).
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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
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Patients with an anticipated survival of at least 3 years in the opinion of the Investigator.
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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.
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Ability to swallow oral medication and be willing to adhere to the study intervention regimen.
Exclusion Criteria:
- 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.
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: Zufällig
- Interventionsmodell: Parallele Zuordnung
- Maskierung: Verdreifachen
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
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Placebo-Komparator: Placebo-Arm
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Oral administration of placebo once daily for 24 weeks in the core part and for a total of 32 weeks including the extension part; Transition to low-dose of ONO-2808 for remainder of the extension part until week 80 and for up to an additional 72 weeks in the additional extension part |
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Experimental: ONO-2808 Arm
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Oral administration of ONO-2808 at low, middle or high doses once daily for 24 weeks in the core part, for a total of 80 weeks including the extension part, and for up to an additional 72 weeks in the additional extension part |
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Incidence of Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Zeitfenster: From screening up to follow-up for the core part (up to Week 28)
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A TEAE was defined as any adverse event occurring or worsening on or after the first dose of study treatment. A TEAE was considered study treatment-related if there was a reasonable causal relationship between study intervention administration and the TEAE. A serious adverse event (SAE) was an event that resulted in death, was life-threatening, required hospitalization or prolongation of hospitalization, resulted in significant disability/incapacity, was a congenital anomaly/birth defect, or was considered an important medical event. |
From screening up to follow-up for the core part (up to Week 28)
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Incidence of TEAEs by Highest Severity
Zeitfenster: From screening up to follow-up for the core part (up to Week 28)
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A TEAE was defined as any adverse event occurring or worsening on or after the first dose of study treatment. |
From screening up to follow-up for the core part (up to Week 28)
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Incidence of Study Treatment-related TEAEs by Highest Severity
Zeitfenster: From screening up to follow-up for the core part (up to Week 28)
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A TEAE was defined as any adverse event occurring or worsening on or after the first dose of study treatment. A TEAE was considered study treatment-related if there was a reasonable causal relationship between study intervention administration and the TEAE. |
From screening up to follow-up for the core part (up to Week 28)
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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Plasma Concentration of ONO-2808
Zeitfenster: Week 2, Week 8, Week 12, and Week 24
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Descriptive summary statistics were calculated for ONO-2808 plasma concentrations, by dose level and time point. |
Week 2, Week 8, Week 12, and Week 24
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Mitarbeiter und Ermittler
Sponsor
Ermittler
- Studienleiter: Project Leader, Ono Pharmaceutical Co., Ltd.
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Primärer Abschluss (Tatsächlich)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Zusätzliche relevante MeSH-Bedingungen
Andere Studien-ID-Nummern
- ONO-2808-03
- jRCT2041230153 (Registrierungskennung: Japan Registry of Clinical Trials)
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