En fase 2-studie av ONO-2808 hos pasienter med multippel systematrofi
En fase 2, dobbeltblind, placebokontrollert, parallellgruppestudie for å vurdere sikkerhet, tolerabilitet, farmakokinetikk, farmakodynamikk og potensiell effekt av flere doser av ONO-2808 hos pasienter med multippel systematrofi (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.
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Faktiske)
Fase
- Fase 2
Kontakter og plasseringer
Studiesteder
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California
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Fountain Valley, California, Forente stater, 92708
- The Parkinson's Movement and Disorder Institute
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Los Angeles, California, Forente stater, 90033
- University of Southern California
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Los Angeles, California, Forente stater, 90095
- David Geffen School of Medicine at UCLA
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Palo Alto, California, Forente stater, 94304
- Stanford University School of Medicine
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Colorado
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Englewood, Colorado, Forente stater, 80113
- CenExel Rocky Mountain Clinical Research
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Connecticut
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New Haven, Connecticut, Forente stater, 06519
- Yale School of Medicine - Yale Church Street Research Unit (CRSU)
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Florida
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Boca Raton, Florida, Forente stater, 33486
- Parkinson's Disease and Movement Disorders Center of Boca Raton
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Gainesville, Florida, Forente stater, 32608
- Norman Fixel Institute for Neurological Diseases - University of Florida
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Jacksonville, Florida, Forente stater, 32224
- Mayo Clinic in Florida
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Miami, Florida, Forente stater, 33136
- University of Miami Miller School of Medicine
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Georgia
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Atlanta, Georgia, Forente stater, 30329
- Emory University School of Medicine
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Kansas
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Kansas City, Kansas, Forente stater, 66160
- University of Kansas Medical Center Research Institute
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Massachusetts
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Boston, Massachusetts, Forente stater, 02115
- Brigham and Women's Hospital
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Boston, Massachusetts, Forente stater, 02114
- Massachusetts General Hospital
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Michigan
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Ann Arbor, Michigan, Forente stater, 48109
- University of Michigan School of Medicine
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Farmington Hills, Michigan, Forente stater, 48334
- Quest Research Institute
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Minnesota
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Rochester, Minnesota, Forente stater, 55905
- Mayo Clinic - Rochester
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Nebraska
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Omaha, Nebraska, Forente stater, 68198
- University of Nebraska Medical Center
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New York
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New York, New York, Forente stater, 10032
- Columbia University
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New York, New York, Forente stater, 10019
- Icahn School of Medicine at Mount Sinai
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New York, New York, Forente stater, 10016
- NYU Langone Health - NYU Dysautonomia Center
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Ohio
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Cincinnati, Ohio, Forente stater, 45219
- University of Cincinnati College of Medicine
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Cleveland, Ohio, Forente stater, 44195
- Cleveland Clinic
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Columbus, Ohio, Forente stater, 43201
- Ohio State University
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Pennsylvania
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Hershey, Pennsylvania, Forente stater, 17033
- Penn State University - Milton S. Hershey Medical Center
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Philadelphia, Pennsylvania, Forente stater, 19107
- The University of Pennsylvania - Pennsylvania Hospital
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Texas
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Dallas, Texas, Forente stater, 75390
- UT Southwestern Medical Center
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Virginia
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Richmond, Virginia, Forente stater, 23298
- Virginia Commonwealth University Medical Center
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Washington
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Kirkland, Washington, Forente stater, 98034
- Evergreen Health
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Seattle, Washington, Forente stater, 98122
- Swedish Neuroscience Institute, Movement Disorders Clinic
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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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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
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.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Trippel
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Placebo komparator: Placeboarm
|
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 |
|
Eksperimentell: 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 |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Incidence of Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Tidsramme: From screening up to follow-up for the core part (up to Week 28)
|
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)
|
|
Incidence of TEAEs by Highest Severity
Tidsramme: From screening up to follow-up for the core part (up to Week 28)
|
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)
|
|
Incidence of Study Treatment-related TEAEs by Highest Severity
Tidsramme: From screening up to follow-up for the core part (up to Week 28)
|
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)
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Plasma Concentration of ONO-2808
Tidsramme: Week 2, Week 8, Week 12, and Week 24
|
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
|
Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Project Leader, Ono Pharmaceutical Co., Ltd.
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Faktiske)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- ONO-2808-03
- jRCT2041230153 (Registeridentifikator: Japan Registry of Clinical Trials)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
Legemiddel- og utstyrsinformasjon, studiedokumenter
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