Studie 2. fáze ONO-2808 u pacientů s mnohočetnou systémovou atrofií
Dvojitě zaslepená, placebem kontrolovaná studie s paralelními skupinami fáze 2 k posouzení bezpečnosti, snášenlivosti, farmakokinetiky, farmakodynamiky a potenciální účinnosti více dávek ONO-2808 u pacientů s mnohočetnou systémovou atrofií (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.
Přehled studie
Postavení
Podmínky
Intervence / Léčba
Detailní popis
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.
Typ studie
Zápis (Aktuální)
Fáze
- Fáze 2
Kontakty a umístění
Studijní místa
-
-
Aichi-ken
-
Toyoake, Aichi-ken, Japonsko
- Fujita Health University Hospital
-
-
Kyoto
-
Kyoto, Kyoto, Japonsko
- National Hospital Organization Utano National Hospital
-
-
Miyagi
-
Sendai, Miyagi, Japonsko
- National Hospital Organization Sendai Nishitaga Hospital
-
-
Osaka
-
Toyonaka, Osaka, Japonsko
- National Hospital Organization Osaka Toneyama Medical Center
-
-
Tokyo
-
Fuchū, Tokyo, Japonsko
- Tokyo Metropolitan Neurological Hospital
-
-
-
-
California
-
Fountain Valley, California, Spojené státy, 92708
- The Parkinson's Movement and Disorder Institute
-
Los Angeles, California, Spojené státy, 90033
- University of Southern California
-
Los Angeles, California, Spojené státy, 90095
- David Geffen School of Medicine at UCLA
-
Palo Alto, California, Spojené státy, 94304
- Stanford University School of Medicine
-
-
Colorado
-
Englewood, Colorado, Spojené státy, 80113
- CenExel Rocky Mountain Clinical Research
-
-
Connecticut
-
New Haven, Connecticut, Spojené státy, 06519
- Yale School of Medicine - Yale Church Street Research Unit (CRSU)
-
-
Florida
-
Boca Raton, Florida, Spojené státy, 33486
- Parkinson's Disease and Movement Disorders Center of Boca Raton
-
Gainesville, Florida, Spojené státy, 32608
- Norman Fixel Institute for Neurological Diseases - University of Florida
-
Jacksonville, Florida, Spojené státy, 32224
- Mayo Clinic in Florida
-
Miami, Florida, Spojené státy, 33136
- University of Miami Miller School of Medicine
-
-
Georgia
-
Atlanta, Georgia, Spojené státy, 30329
- Emory University School of Medicine
-
-
Kansas
-
Kansas City, Kansas, Spojené státy, 66160
- University of Kansas Medical Center Research Institute
-
-
Massachusetts
-
Boston, Massachusetts, Spojené státy, 02115
- Brigham and Women's Hospital
-
Boston, Massachusetts, Spojené státy, 02114
- Massachusetts General Hospital
-
-
Michigan
-
Ann Arbor, Michigan, Spojené státy, 48109
- University of Michigan School of Medicine
-
Farmington Hills, Michigan, Spojené státy, 48334
- Quest Research Institute
-
-
Minnesota
-
Rochester, Minnesota, Spojené státy, 55905
- Mayo Clinic - Rochester
-
-
Nebraska
-
Omaha, Nebraska, Spojené státy, 68198
- University of Nebraska Medical Center
-
-
New York
-
New York, New York, Spojené státy, 10032
- Columbia University
-
New York, New York, Spojené státy, 10019
- Icahn School of Medicine at Mount Sinai
-
New York, New York, Spojené státy, 10016
- NYU Langone Health - NYU Dysautonomia Center
-
-
Ohio
-
Cincinnati, Ohio, Spojené státy, 45219
- University of Cincinnati College of Medicine
-
Cleveland, Ohio, Spojené státy, 44195
- Cleveland Clinic
-
Columbus, Ohio, Spojené státy, 43201
- Ohio State University
-
-
Pennsylvania
-
Hershey, Pennsylvania, Spojené státy, 17033
- Penn State University - Milton S. Hershey Medical Center
-
Philadelphia, Pennsylvania, Spojené státy, 19107
- The University of Pennsylvania - Pennsylvania Hospital
-
-
Texas
-
Dallas, Texas, Spojené státy, 75390
- UT Southwestern Medical Center
-
-
Virginia
-
Richmond, Virginia, Spojené státy, 23298
- Virginia Commonwealth University Medical Center
-
-
Washington
-
Kirkland, Washington, Spojené státy, 98034
- Evergreen Health
-
Seattle, Washington, Spojené státy, 98122
- Swedish Neuroscience Institute, Movement Disorders Clinic
-
-
Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
Přijímá zdravé dobrovolníky
Pohlaví způsobilá ke studiu
Popis
Inclusion Criteria:
-
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 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.
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.
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Paralelní přiřazení
- Maskování: Trojnásobný
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
|
Komparátor placeba: Placebo Arm
|
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 |
|
Experimentální: ONO-2808 Arm
|
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 |
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Incidence of Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Časové okno: 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
Časové okno: 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
Časové okno: 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)
|
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Plasma Concentration of ONO-2808
Časové okno: 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
|
Spolupracovníci a vyšetřovatelé
Sponzor
Vyšetřovatelé
- Ředitel studie: Project Leader, Ono Pharmaceutical Co., Ltd.
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Aktuální)
Primární dokončení (Aktuální)
Dokončení studie (Odhadovaný)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
Další identifikační čísla studie
- ONO-2808-03
- jRCT2041230153 (Identifikátor registru: Japan Registry of Clinical Trials)
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .