A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
2019년 5월 7일 업데이트: Cytokinetics
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)
The primary objective of this study is to demonstrate a pharmacodynamic effect of CK 2017357 on measures of skeletal muscle function or fatigability in patients with ALS.
연구 개요
상세 설명
This study is a Phase II, double-blind, randomized, placebo-controlled, three-way crossover study of CK-2017357 in patients with ALS.
36 to 72 patients will be randomized to one of six different treatment sequences.
Each treatment sequence consists of three dosing periods; in each dosing period¸ patients receive a single oral dose of placebo, 250 mg of CK-2017357, or 500 mg of CK-2017357.
All six treatment sequences will enroll approximately the same number of patients.
A washout period of at least 6 days (to a maximum of 10 days) will be employed between the doses for each patient.
This study is designed to assess the effect of CK-2017357 on maximal voluntary muscle strength, on the development of fatigue at maximal and sub-maximal voluntary muscle contraction, and on selected pulmonary function parameters.
The plasma concentration of CK-2017357 will be measured at selected time points after each of two single doses of CK-2017357 in men and women.
The plasma concentration versus time data obtained in this study may be used to develop a population PK model and estimate inter-subject variability of PK parameters in this target patient population, in particular between male and female study patients.
연구 유형
중재적
등록 (실제)
67
단계
- 2 단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 장소
-
-
Arizona
-
Phoenix, Arizona, 미국, 85018
- Phoenix Neurological Associates, Ltd.
-
-
California
-
Fresno, California, 미국, 93701
- University Neurology Associates
-
San Francisco, California, 미국, 94115
- California Pacific Medical Center
-
-
Florida
-
Jacksonville, Florida, 미국, 32224
- Mayo Clinic Florida
-
-
Kentucky
-
Lexington, Kentucky, 미국, 40536
- University of Kentucky
-
-
Maryland
-
Baltimore, Maryland, 미국, 21287
- Johns Hopkins Hospital
-
-
Massachusetts
-
Boston, Massachusetts, 미국, 02114
- Massachusetts General Hospital
-
-
Missouri
-
Saint Louis, Missouri, 미국, 63110
- Washington University
-
-
New York
-
Syracuse, New York, 미국, 13210
- SUNY Upstate Medical Center
-
-
North Carolina
-
Durham, North Carolina, 미국, 27705
- Duke University
-
-
Oregon
-
Portland, Oregon, 미국, 97213
- Providence ALS Center
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, 미국, 19102
- Drexel University College of Medicine, Dept of Neurology
-
University Park, Pennsylvania, 미국, 17033
- Penn State
-
-
Texas
-
San Antonio, Texas, 미국, 78229
- The University of Texas Health Science Center at San Antonio
-
-
Vermont
-
Burlington, Vermont, 미국, 05401
- University of Vermont
-
-
참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
18년 이상 (성인, 고령자)
건강한 자원 봉사자를 받아들입니다
아니
연구 대상 성별
모두
설명
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
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 크로스오버 할당
- 마스킹: 네 배로
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: 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.
|
단일 경구 용량으로 투여되는 캡슐의 일치하는 위약.
CK-2017357 캡슐 250mg 단일 경구 투여.
다른 이름들:
CK-2017357 캡슐 500mg 단일 경구 투여.
다른 이름들:
|
|
실험적: 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.
|
단일 경구 용량으로 투여되는 캡슐의 일치하는 위약.
CK-2017357 캡슐 250mg 단일 경구 투여.
다른 이름들:
CK-2017357 캡슐 500mg 단일 경구 투여.
다른 이름들:
|
|
실험적: 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.
|
단일 경구 용량으로 투여되는 캡슐의 일치하는 위약.
CK-2017357 캡슐 250mg 단일 경구 투여.
다른 이름들:
CK-2017357 캡슐 500mg 단일 경구 투여.
다른 이름들:
|
|
실험적: 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.
|
단일 경구 용량으로 투여되는 캡슐의 일치하는 위약.
CK-2017357 캡슐 250mg 단일 경구 투여.
다른 이름들:
CK-2017357 캡슐 500mg 단일 경구 투여.
다른 이름들:
|
|
실험적: 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.
|
단일 경구 용량으로 투여되는 캡슐의 일치하는 위약.
CK-2017357 캡슐 250mg 단일 경구 투여.
다른 이름들:
CK-2017357 캡슐 500mg 단일 경구 투여.
다른 이름들:
|
|
실험적: 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.
|
단일 경구 용량으로 투여되는 캡슐의 일치하는 위약.
CK-2017357 캡슐 250mg 단일 경구 투여.
다른 이름들:
CK-2017357 캡슐 500mg 단일 경구 투여.
다른 이름들:
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
ALSFRS-R
기간: 2 days
|
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.
|
2 days
|
|
Maximum grip strength
기간: 2 days
|
Measured using the DynEx Electronic Hand Dynamometer.
Patients asked to squeeze the device with the maximum possible force to establish the maximum voluntary contraction.
|
2 days
|
|
Maximum grip strength fatigability
기간: 2 days
|
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.
|
2 days
|
|
Shoulder extension fatigue
기간: 2 days
|
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.
|
2 days
|
|
Slow Vital Capacity (SVC)
기간: 2 days
|
SVC is measured using the Puritan Bennett Renaissance II Spirometry System and accessories.
|
2 days
|
|
Maximum Voluntary Ventilation (MVV)
기간: 2 days
|
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.
|
2 days
|
|
Sniff Inspiratory Pressure (SNIP)
기간: 2 days
|
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
|
2 days
|
|
Maximum Voluntary Muscle Contraction (MVC)
기간: 2 days
|
MVC is measured using the MicroFET 2 HHD.
|
2 days
|
|
Repeated Sub-Maximum Grip Strength Fatigability
기간: 2 days
|
Sub-Maximum Grip Strength Fatigability is measured using the DynEx Electronic Hand.
Dynamometer
|
2 days
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
부작용이 있는 환자 수
기간: 4 주
|
4 주
|
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and ALSFRS-R.
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength fatigability
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and shoulder extension fatigue
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and slow vital capacity
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary ventilation
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and sniff inspiratory pressure
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary muscle contraction
기간: 2 days
|
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.
|
2 days
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and repeated sub-maximum grip strength fatigability
기간: 2 days
|
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.
|
2 days
|
|
Effect of CK-2017357 on patient determined global functional assessment
기간: 2 days
|
Patients will be asked to assess whether they feel the same, better or worse as compared to how they felt pre-dose
|
2 days
|
|
Effect of CK-2017357 on investigator determined global functional assessment
기간: 2 days
|
Investigator will assess whether they the patient appears the same, better or worse as compared to the patient's status at pre-dose
|
2 days
|
공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
스폰서
수사관
- 연구 의자: Jeremy M Shefner, MD, PhD, State University of New York - Upstate Medical University
간행물 및 유용한 링크
연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작
2010년 3월 1일
기본 완료 (실제)
2010년 11월 1일
연구 완료 (실제)
2010년 11월 1일
연구 등록 날짜
최초 제출
2010년 3월 16일
QC 기준을 충족하는 최초 제출
2010년 3월 17일
처음 게시됨 (추정)
2010년 3월 18일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2019년 5월 10일
QC 기준을 충족하는 마지막 업데이트 제출
2019년 5월 7일
마지막으로 확인됨
2019년 5월 1일
추가 정보
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .
근위축성 측삭 경화증에 대한 임상 시험
-
Sanko University완전한MULTİPLE SCLEROSİS | BALANCE | 유효성 | 신뢰도터키 (Türkiye)
위약에 대한 임상 시험
-
Newish Biotech (Wuxi) Co., Ltd.아직 모집하지 않음
-
Chiesi Farmaceutici S.p.A.아직 모집하지 않음
-
Nature's Sunshine Products, Inc.아직 모집하지 않음
-
Yale UniversityHartford HealthCare아직 모집하지 않음
-
Acesion Pharma모병심방세동(AF)헝가리, 폴란드, 불가리아, 덴마크, 독일, 네덜란드, 이탈리아, 세르비아
-
Shanghai Lanyi Therapeutics Co., Ltd.완전한
-
University of Texas Southwestern Medical Center아직 모집하지 않음
-
Universidad Autonoma de Zacatecas모집하지 않고 적극적으로