Methodology Study of Novel Outcome Measures to Assess Progression of ALS
2019년 10월 23일 업데이트: Biogen
Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis
The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of outcome measures administered to participants with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
The secondary objectives of this study are: To evaluate the test-retest reproducibility of each outcome measure; To determine correlations between 6 and 12-month changes in all exploratory measures with 18 and 24-month changes in ALSFRS-R and survival; To assess correlations between/among the various measures; To obtain biological samples in order to identify molecular correlates to the clinical measures and to further characterize previously identified and novel molecular biomarkers of disease progression for incorporation into future clinical studies.
연구 개요
상태
완전한
정황
연구 유형
관찰
등록 (실제)
138
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 장소
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CX
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Utrecht, CX, 네덜란드, 3584
- UMC Utrecht
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Berlin, 독일, 13125
- Charite - Campus Virchow-Klinikum
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Hannover, 독일, 30625
- Medizinische Hochschule Hannover
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Jena, 독일, 07743
- Universitaetsklinikum Jena
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Ulm, 독일, 89081
- Universitaetsklinikum Ulm
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California
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San Diego, California, 미국, 92103
- University of California San Diego Medical Center
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San Francisco, California, 미국, 94115
- California Pacific Medical Center
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Florida
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Tampa, Florida, 미국, 33612
- University of South Florida
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Georgia
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Atlanta, Georgia, 미국, 30322
- The Emory Clinic
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Maryland
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Baltimore, Maryland, 미국, 21287
- Johns Hopkins Hospital
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Massachusetts
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Charlestown, Massachusetts, 미국, 2129
- Massachusetts General Hospital, MA
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Missouri
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Saint Louis, Missouri, 미국, 63110
- Washington University School of Medicine
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Pennsylvania
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Hershey, Pennsylvania, 미국, EC037
- Penn State Milton S. Hershey Medical Center
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Leuven, 벨기에, 3000
- UZ Leuven
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St. Gallen, 스위스, 9007
- Kantonsspital St. Gallen
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Dublin, 아일랜드, Dublin 9
- Beaumont Hospital
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West Midlands
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Sheffield, West Midlands, 영국, S102JF
- Royal Hallamshire Hospital
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Ontario
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Toronto, Ontario, 캐나다, M4N 3M5
- Sunnybrook Health Sciences Centre
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Quebec
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Montréal, Quebec, 캐나다, H3A 2B4
- Montreal Neurological Institute Clinical Research Unit
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Hérault
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Montpellier, Hérault, 프랑스, 34295
- Hopital Gui de Chauliac, Service de Neurologie
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Paris
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Paris cedex 13, Paris, 프랑스, 75013
- Groupe Hospitalier Pitie-Salpetriere
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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
16년 (어린이, 성인, 고령자)
건강한 자원 봉사자를 받아들입니다
아니
연구 대상 성별
모두
샘플링 방법
확률 샘플
연구 인구
Participants suffering from ALS are recruited by participating physicians in a standard clinical practice setting.
설명
Key Inclusion Criteria:
- A diagnosis of sporadic or familial ALS
- ALS onset within ≤5 years
- Must be 16 to 85 years of age, inclusive, for sites in the United States and 18 to 85 years of age, inclusive, for all sites outside of the United States
Key Exclusion Criteria:
- History of or positive test result at Screening for human immunodeficiency virus (HIV)
- History of or positive test result at Screening for hepatitis C virus (HCV) antibody or hepatitis B virus (HBV)
- Possibility of neuromuscular weakness other than ALS
- Unspecified reasons that, in the opinion of the site Investigator, make the subject unsuitable for enrollment or unlikely to be able to complete, at a minimum, the Month 6 Visit
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM)
기간: Baseline to Month 6 and Baseline to Month 12
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EIM is an electrophysiological technique in which current is applied to a muscle of interest and resultant voltage and impedance are measured.
These measured parameters reflect the conductivity of underlying tissue and presumably the pathologic state of denervated muscle in an ALS participant
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in electrophysiological measures as assessed by compound muscle action potential (CMAP)
기간: Baseline to Month 6 and Baseline to Month 12
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CMAP is a standard electrophysiological measure generated by maximally stimulating a nerve such that all muscle fibers innervated by the respective nerve are depolarized.
Reduction of CMAP amplitude reflects loss of motor axons and, therefore, is directly relevant to ALS.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number estimation (MUNE)
기간: Baseline to Month 6 and Baseline to Month 12
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Optional, to be administered at each site's Investigator's discretion.
MUNE is used to estimate the number of functioning motor units.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number index (MUNIX)
기간: Baseline to Month 6 and Baseline to Month 12
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MUNIX estimates functioning motor units within a muscle.
CMAP and surface electromyography potentials (surface interference patterns) are obtained at various levels of voluntary effort, and MUNIX is estimated using power and area of CMAP and surface interference patterns.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in muscle strength measures as assessed by hand-held dynamometry (HHD)
기간: Baseline to Month 6 and Baseline to Month 12
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HHD tests isometric strength of multiple muscles using standard participant positioning.
Approximately 10 muscle groups will be examined (per each side) in both upper and lower extremities.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in respiratory measures as assessed by slow vital capacity (SVC)
기간: Baseline to Month 6 and Baseline to Month 12
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Vital capacity will be measured by means of an SVC test, administered in the upright position.
Upright SVC will be determined by performing 3 to 5 measures, in accordance with criteria established by the American Thoracic Society and the European Respiratory Society.
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Baseline to Month 6 and Baseline to Month 12
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Longitudinal standardized mean change in functional measures as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
기간: Baseline to Month 6 and Baseline to Month 12
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The ALSFRS-R has been demonstrated to predict survival.
The ALSFRS-R measures 4 functional domains, including respiratory, bulbar function, gross motor skills, and fine motor skills.
There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48 [Cedarbaum 1999], with higher scores representing better function.
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Baseline to Month 6 and Baseline to Month 12
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2차 결과 측정
결과 측정 |
기간 |
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Within-participant test-retest reliability between the 2 repeated measurements occurring on Day 1 and Day 7 for EIM
기간: Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for CMAP
기간: Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for MUNE
기간: Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for MUNIX
기간: Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for HHD
기간: Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for SVC
기간: Day 1 and Day 7
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Day 1 and Day 7
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Within-participant test-retest reliability between the 2 repeated measurements for ALSFRS-R
기간: Day 1 and Day 7
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Day 1 and Day 7
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Comparison between 6 and 12-month changes in exploratory measures with 18 and 24-month changes in ALSFRS-R and survival
기간: Baseline to Month 24
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Baseline to Month 24
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Comparison between 6-month changes for muscle electrophysiological measures
기간: Baseline to Month 12
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Baseline to Month 12
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Comparison between 6-month changes for muscle strength measures
기간: Baseline to Month 12
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Baseline to Month 12
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Comparison between 6-month changes for functional measures
기간: Baseline to Month 12
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Baseline to Month 12
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Comparison of molecular biomarkers with disease progression
기간: Baseline to Month 12
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Baseline to Month 12
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
스폰서
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 (실제)
2016년 1월 6일
기본 완료 (실제)
2018년 7월 27일
연구 완료 (실제)
2019년 8월 1일
연구 등록 날짜
최초 제출
2015년 10월 8일
QC 기준을 충족하는 최초 제출
2015년 11월 19일
처음 게시됨 (추정)
2015년 11월 23일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2019년 10월 24일
QC 기준을 충족하는 마지막 업데이트 제출
2019년 10월 23일
마지막으로 확인됨
2019년 10월 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. .