Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

Methodology Study of Novel Outcome Measures to Assess Progression of ALS

23. oktober 2019 opdateret af: 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.

Studieoversigt

Status

Afsluttet

Betingelser

Undersøgelsestype

Observationel

Tilmelding (Faktiske)

138

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

16 år til 85 år (Barn, Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Prøveudtagningsmetode

Sandsynlighedsprøve

Studiebefolkning

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

Beskrivelse

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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

Sekundære resultatmål

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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Biogen

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

6. januar 2016

Primær færdiggørelse (Faktiske)

27. juli 2018

Studieafslutning (Faktiske)

1. august 2019

Datoer for studieregistrering

Først indsendt

8. oktober 2015

Først indsendt, der opfyldte QC-kriterier

19. november 2015

Først opslået (Skøn)

23. november 2015

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

24. oktober 2019

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

23. oktober 2019

Sidst verificeret

1. oktober 2019

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • 999AS003

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Amyotrofisk lateral sklerose

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in India

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner