- ICH GCP
- Реестр клинических исследований США
- Клиническое испытание NCT01089010
A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase II, Double-Blind, Randomized, Placebo-Controlled, Three-Way Crossover, Pharmacokinetic and Pharmacodynamic Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Обзор исследования
Статус
Условия
Вмешательство/лечение
Подробное описание
Тип исследования
Регистрация (Действительный)
Фаза
- Фаза 2
Контакты и местонахождение
Места учебы
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Arizona
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Phoenix, Arizona, Соединенные Штаты, 85018
- Phoenix Neurological Associates, Ltd.
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California
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Fresno, California, Соединенные Штаты, 93701
- University Neurology Associates
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San Francisco, California, Соединенные Штаты, 94115
- California Pacific Medical Center
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Florida
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Jacksonville, Florida, Соединенные Штаты, 32224
- Mayo Clinic Florida
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Kentucky
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Lexington, Kentucky, Соединенные Штаты, 40536
- University of Kentucky
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Maryland
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Baltimore, Maryland, Соединенные Штаты, 21287
- Johns Hopkins Hospital
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Massachusetts
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Boston, Massachusetts, Соединенные Штаты, 02114
- Massachusetts General Hospital
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Missouri
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Saint Louis, Missouri, Соединенные Штаты, 63110
- Washington University
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New York
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Syracuse, New York, Соединенные Штаты, 13210
- SUNY Upstate Medical Center
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North Carolina
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Durham, North Carolina, Соединенные Штаты, 27705
- Duke University
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Oregon
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Portland, Oregon, Соединенные Штаты, 97213
- Providence ALS Center
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Pennsylvania
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Philadelphia, Pennsylvania, Соединенные Штаты, 19102
- Drexel University College of Medicine, Dept of Neurology
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University Park, Pennsylvania, Соединенные Штаты, 17033
- Penn State
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Texas
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San Antonio, Texas, Соединенные Штаты, 78229
- The University of Texas Health Science Center at San Antonio
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Vermont
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Burlington, Vermont, Соединенные Штаты, 05401
- University of Vermont
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Критерии участия
Критерии приемлемости
Возраст, подходящий для обучения
Принимает здоровых добровольцев
Полы, имеющие право на обучение
Описание
Inclusion Criteria
For enrollment, patients were required to satisfy all of the following criteria at baseline:
1. Able to comprehend and willing to sign an Informed Consent Form (ICF)
- A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) (Brooks, Miller et al. 2000)
- Males or females 18 years of age or older
- Body Mass Index (BMI) of 18.0 to 30.0 kg/m2, inclusive
- Maximum voluntary grip strength in at least one hand between 10 and 40 pounds (females) or 10 and 60 pounds (males)
- Able to swallow capsules with water
- Upright Slow Vital Capacity (SVC) > 40% of predicted for age, height, and sex [See Appendix 16.6.1]
- Able to perform pulmonary function tests
- Pre-study clinical laboratory findings (including troponin I [TnI] and creatine phosphokinase [CPK]) within normal range, or, if outside of the normal range, deemed not clinically significant by the Investigator
- For female patients only: The patient is post-menopausal (≥ 1 year) or sterilized, or if she is of childbearing potential, she is not breastfeeding, her pregnancy test is negative, she has no intention to become pregnant during the course of the study, and she is using contraceptive drugs or devices for the duration of the study and for 10 weeks after the end of the study.
For male patients only: Male patients agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of reproductive potential and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide or oral contraceptives) or the male patient must agree to abstain from sexual intercourse for 10 weeks after the end of the study.
Exclusion Criteria
Patients satisfying any of the following criteria at baseline were excluded from enrollment:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper limit of normal (ULN)
- Life expectancy < 3 months
- Participation in any trial in which receipt of investigational study drug occurred within 30 days prior to dosing
- Any prior treatment with CK-2017357
- In the opinion of the Investigator, the patient is not suitable to participate in the study
Учебный план
Как устроено исследование?
Детали дизайна
- Основная цель: Уход
- Распределение: Рандомизированный
- Интервенционная модель: Назначение кроссовера
- Маскировка: Четырехместный
Оружие и интервенции
Группа участников / Армия |
Вмешательство/лечение |
---|---|
Экспериментальный: Treatment Sequence 1
Treatment sequence 1 consisted of three dosing periods in which patients received single oral doses of placebo, 250 mg, and 500 mg of CK-2017357, in that order, with approximately one week between each dose.
Each patient acted as their own control, as all doses were represented in each treatment sequence.
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Соответствующее плацебо в капсулах, вводимых однократно перорально.
250 мг CK-2017357 в капсулах, вводимых однократно перорально.
Другие имена:
500 мг CK-2017357 в капсулах вводят однократно перорально.
Другие имена:
|
Экспериментальный: Treatment Sequence 2
Treatment sequence 2 consisted of three dosing periods in which patients received single oral doses of placebo, 500 mg, and 250 mg of CK-2017357, in that order, with approximately one week between each dose.
Each patient acted as their own control, as all doses were represented in each treatment sequence.
|
Соответствующее плацебо в капсулах, вводимых однократно перорально.
250 мг CK-2017357 в капсулах, вводимых однократно перорально.
Другие имена:
500 мг CK-2017357 в капсулах вводят однократно перорально.
Другие имена:
|
Экспериментальный: Treatment Sequence 3
Treatment sequence 3 consisted of three dosing periods in which patients received single oral doses of 250 mg, placebo and 500 mg of CK-2017357, in that order, with approximately one week between each dose.
Each patient acted as their own control, as all doses were represented in each treatment sequence.
|
Соответствующее плацебо в капсулах, вводимых однократно перорально.
250 мг CK-2017357 в капсулах, вводимых однократно перорально.
Другие имена:
500 мг CK-2017357 в капсулах вводят однократно перорально.
Другие имена:
|
Экспериментальный: Treatment Sequence 4
Treatment sequence 4 consisted of three dosing periods in which patients received single oral doses of 250 mg, 500 mg and placebo of CK-2017357, in that order, with approximately one week between each dose.
Each patient acted as their own control, as all doses were represented in each treatment sequence.
|
Соответствующее плацебо в капсулах, вводимых однократно перорально.
250 мг CK-2017357 в капсулах, вводимых однократно перорально.
Другие имена:
500 мг CK-2017357 в капсулах вводят однократно перорально.
Другие имена:
|
Экспериментальный: Treatment Sequence 5
Treatment sequence 5 consisted of three dosing periods in which patients received single oral doses of 500 mg, placebo, and 250 mg of CK-2017357, in that order, with approximately one week between each dose.
Each patient acted as their own control, as all doses were represented in each treatment sequence.
|
Соответствующее плацебо в капсулах, вводимых однократно перорально.
250 мг CK-2017357 в капсулах, вводимых однократно перорально.
Другие имена:
500 мг CK-2017357 в капсулах вводят однократно перорально.
Другие имена:
|
Экспериментальный: Treatment Sequence 6
Treatment sequence6 consisted of three dosing periods in which patients received single oral doses of 500 mg, 250 mg, and placebo of CK-2017357, in that order, with approximately one week between each dose.
Each patient acted as their own control, as all doses were represented in each treatment sequence.
|
Соответствующее плацебо в капсулах, вводимых однократно перорально.
250 мг CK-2017357 в капсулах, вводимых однократно перорально.
Другие имена:
500 мг CK-2017357 в капсулах вводят однократно перорально.
Другие имена:
|
Что измеряет исследование?
Первичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
---|---|---|
ALSFRS-R
Временное ограничение: 2 days
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An instrument for evaluating the functional status of patients with ALS.
Minimum score is 0 and maximum score is 40.
The higher the score the more function is retained.
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2 days
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Maximum grip strength
Временное ограничение: 2 days
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Measured using the DynEx Electronic Hand Dynamometer.
Patients asked to squeeze the device with the maximum possible force to establish the maximum voluntary contraction.
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2 days
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Maximum grip strength fatigability
Временное ограничение: 2 days
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Handgrip fatigue is measured using the DynEx Electronic Hand Dynamometer.
Patient is asked to squeeze the device until they can no longer stay above 60% of target or 120 seconds.
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2 days
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Shoulder extension fatigue
Временное ограничение: 2 days
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Patient is asked to hold one arm outstretched in front of them at a 90 degree angle.
The time the arm falls below 90 degrees for > 2 seconds will be recorded, up to a total evaluation time of 2 minutes.
This is then repeated with the other arm.
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2 days
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Slow Vital Capacity (SVC)
Временное ограничение: 2 days
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SVC is measured using the Puritan Bennett Renaissance II Spirometry System and accessories.
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2 days
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Maximum Voluntary Ventilation (MVV)
Временное ограничение: 2 days
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MVV is the volume of air that can be exhaled during 12 seconds of rapid deep breathing.
The actual volume is extrapolated to one minute.
the Puritan Bennett Renaissance II Spirometry System and accessories is used for this measurement.
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2 days
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Sniff Inspiratory Pressure (SNIP)
Временное ограничение: 2 days
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SNIP is measured at Functional Residual Capacity, the bottom of the tidal breathing cycle, through one plugged nostril while the other remains open using the Micro Medical MicroRPM Respiratory Pressure Meter
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2 days
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Maximum Voluntary Muscle Contraction (MVC)
Временное ограничение: 2 days
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MVC is measured using the MicroFET 2 HHD.
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2 days
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Repeated Sub-Maximum Grip Strength Fatigability
Временное ограничение: 2 days
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Sub-Maximum Grip Strength Fatigability is measured using the DynEx Electronic Hand.
Dynamometer
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2 days
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Вторичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
---|---|---|
Количество пациентов с нежелательными явлениями
Временное ограничение: 4 недели
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4 недели
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and ALSFRS-R.
Временное ограничение: 2 days
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ALSFRS-R assessments will be paired with PK concentrations obtained at or near the same time as the ALSFRS-R assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength
Временное ограничение: 2 days
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Maximum grip strength assessments will be paired with PK concentrations obtained at or near the same time as the maximum grip strength assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength fatigability
Временное ограничение: 2 days
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Maximum grip strength fatigability assessments will be paired with PK concentrations obtained at or near the same time as the maximum grip strength fatigability assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and shoulder extension fatigue
Временное ограничение: 2 days
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Shoulder extension fatigue assessments will be paired with PK concentrations obtained at or near the same time as the shoulder extension fatigue assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and slow vital capacity
Временное ограничение: 2 days
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Slow vital capacity assessments will be paired with PK concentrations obtained at or near the same time as the slow vital capacity assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary ventilation
Временное ограничение: 2 days
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Maximum voluntary ventilation assessments will be paired with PK concentrations obtained at or near the same time as the maximum voluntary ventilation assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and sniff inspiratory pressure
Временное ограничение: 2 days
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Sniff inspiratory pressure assessments will be paired with PK concentrations obtained at or near the same time as the sniff inspiratory pressure assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary muscle contraction
Временное ограничение: 2 days
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Maximum voluntary muscle contraction assessments will be paired with PK concentrations obtained at or near the same time as the maximum voluntary muscle contraction assessments and analyzed for concentration related effects.
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2 days
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Characterize the relationship, if any, between the plasma concentration of CK-2017357 and repeated sub-maximum grip strength fatigability
Временное ограничение: 2 days
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Repeated sub-maximum grip strength fatigability assessments will be paired with PK concentrations obtained at or near the same time as the repeated sub-maximum grip strength fatigability assessments and analyzed for concentration related effects.
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2 days
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Effect of CK-2017357 on patient determined global functional assessment
Временное ограничение: 2 days
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Patients will be asked to assess whether they feel the same, better or worse as compared to how they felt pre-dose
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2 days
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Effect of CK-2017357 on investigator determined global functional assessment
Временное ограничение: 2 days
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Investigator will assess whether they the patient appears the same, better or worse as compared to the patient's status at pre-dose
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2 days
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Соавторы и исследователи
Спонсор
Следователи
- Учебный стул: Jeremy M Shefner, MD, PhD, State University of New York - Upstate Medical University
Публикации и полезные ссылки
Даты записи исследования
Изучение основных дат
Начало исследования
Первичное завершение (Действительный)
Завершение исследования (Действительный)
Даты регистрации исследования
Первый отправленный
Впервые представлено, что соответствует критериям контроля качества
Первый опубликованный (Оценивать)
Обновления учебных записей
Последнее опубликованное обновление (Действительный)
Последнее отправленное обновление, отвечающее критериям контроля качества
Последняя проверка
Дополнительная информация
Термины, связанные с этим исследованием
Дополнительные соответствующие термины MeSH
- Патологические процессы
- Метаболические заболевания
- Заболевания центральной нервной системы
- Заболевания нервной системы
- Нервно-мышечные заболевания
- Нейродегенеративные заболевания
- Заболевания спинного мозга
- TDP-43 Протеинопатии
- Недостатки протеостаза
- Склероз
- Болезнь двигательных нейронов
- Боковой амиотрофический склероз
Другие идентификационные номера исследования
- CY 4021
Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .
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