Denna sida har översatts automatiskt och översättningens korrekthet kan inte garanteras. Vänligen se engelsk version för en källtext.

Methodology Study of Novel Outcome Measures to Assess Progression of ALS

23 oktober 2019 uppdaterad av: 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.

Studieöversikt

Status

Avslutad

Betingelser

Studietyp

Observationell

Inskrivning (Faktisk)

138

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • Belgien
      • Leuven, Belgien, 3000
        • UZ Leuven
  • Frankrike
    • Hérault
      • Montpellier, Hérault, Frankrike, 34295
        • Hopital Gui de Chauliac, Service de Neurologie
    • Paris
      • Paris cedex 13, Paris, Frankrike, 75013
        • Groupe Hospitalier Pitie-Salpetriere
  • Förenta staterna
    • California
      • San Diego, California, Förenta staterna, 92103
        • University of California San Diego Medical Center
      • San Francisco, California, Förenta staterna, 94115
        • California Pacific Medical Center
    • Florida
      • Tampa, Florida, Förenta staterna, 33612
        • University of South Florida
    • Georgia
      • Atlanta, Georgia, Förenta staterna, 30322
        • The Emory Clinic
    • Maryland
      • Baltimore, Maryland, Förenta staterna, 21287
        • Johns Hopkins Hospital
    • Massachusetts
      • Charlestown, Massachusetts, Förenta staterna, 2129
        • Massachusetts General Hospital, MA
    • Missouri
      • Saint Louis, Missouri, Förenta staterna, 63110
        • Washington University School of Medicine
    • Pennsylvania
      • Hershey, Pennsylvania, Förenta staterna, EC037
        • Penn State Milton S. Hershey Medical Center
  • Irland
      • Dublin, Irland, Dublin 9
        • Beaumont Hospital
  • Kanada
    • Ontario
      • Toronto, Ontario, Kanada, M4N 3M5
        • Sunnybrook Health Sciences Centre
    • Quebec
      • Montréal, Quebec, Kanada, H3A 2B4
        • Montreal Neurological Institute Clinical Research Unit
  • Nederländerna
    • CX
      • Utrecht, CX, Nederländerna, 3584
        • UMC Utrecht
  • Schweiz
      • St. Gallen, Schweiz, 9007
        • Kantonsspital St. Gallen
  • Storbritannien
    • West Midlands
      • Sheffield, West Midlands, Storbritannien, S102JF
        • Royal Hallamshire Hospital
  • Tyskland
      • Berlin, Tyskland, 13125
        • Charite - Campus Virchow-Klinikum
      • Hannover, Tyskland, 30625
        • Medizinische Hochschule Hannover
      • Jena, Tyskland, 07743
        • Universitaetsklinikum Jena
      • Ulm, Tyskland, 89081
        • Universitaetsklinikum Ulm

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

16 år till 85 år (Barn, Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Testmetod

Sannolikhetsprov

Studera befolkning

Participants suffering from ALS are recruited by participating physicians in a standard clinical practice setting.

Beskrivning

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

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM)
Tidsram: Baseline to Month 6 and Baseline to Month 12
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
Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in electrophysiological measures as assessed by compound muscle action potential (CMAP)
Tidsram: Baseline to Month 6 and Baseline to Month 12
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.
Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number estimation (MUNE)
Tidsram: Baseline to Month 6 and Baseline to Month 12
Optional, to be administered at each site's Investigator's discretion. MUNE is used to estimate the number of functioning motor units.
Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number index (MUNIX)
Tidsram: Baseline to Month 6 and Baseline to Month 12
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.
Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in muscle strength measures as assessed by hand-held dynamometry (HHD)
Tidsram: Baseline to Month 6 and Baseline to Month 12
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.
Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in respiratory measures as assessed by slow vital capacity (SVC)
Tidsram: Baseline to Month 6 and Baseline to Month 12
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.
Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in functional measures as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Tidsram: Baseline to Month 6 and Baseline to Month 12
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.
Baseline to Month 6 and Baseline to Month 12

Sekundära resultatmått

Resultatmått
Tidsram
Within-participant test-retest reliability between the 2 repeated measurements occurring on Day 1 and Day 7 for EIM
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for CMAP
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for MUNE
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for MUNIX
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for HHD
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for SVC
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for ALSFRS-R
Tidsram: Day 1 and Day 7
Day 1 and Day 7
Comparison between 6 and 12-month changes in exploratory measures with 18 and 24-month changes in ALSFRS-R and survival
Tidsram: Baseline to Month 24
Baseline to Month 24
Comparison between 6-month changes for muscle electrophysiological measures
Tidsram: Baseline to Month 12
Baseline to Month 12
Comparison between 6-month changes for muscle strength measures
Tidsram: Baseline to Month 12
Baseline to Month 12
Comparison between 6-month changes for functional measures
Tidsram: Baseline to Month 12
Baseline to Month 12
Comparison of molecular biomarkers with disease progression
Tidsram: Baseline to Month 12
Baseline to Month 12

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Biogen

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

6 januari 2016

Primärt slutförande (Faktisk)

27 juli 2018

Avslutad studie (Faktisk)

1 augusti 2019

Studieregistreringsdatum

Först inskickad

8 oktober 2015

Först inskickad som uppfyllde QC-kriterierna

19 november 2015

Första postat (Uppskatta)

23 november 2015

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

24 oktober 2019

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

23 oktober 2019

Senast verifierad

1 oktober 2019

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • 999AS003

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

Kliniska prövningar på Amyotrofisk lateral skleros

Sök liknande försök

Sponsorer och medarbetare

Medicinska tillstånd

Läkemedelsinterventioner

CROs by country

CROs in Bahrain

Betingelser

Sällsynta sjukdomar

Läkemedelsinterventioner

Kosttillskott

Sponsor / medarbetare

Platser

3
Prenumerera