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Methodology Study of Novel Outcome Measures to Assess Progression of ALS

23. oktober 2019 oppdatert 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.

Studieoversikt

Status

Fullført

Forhold

Studietype

Observasjonsmessig

Registrering (Faktiske)

138

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Belgia
      • Leuven, Belgia, 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
  • Forente stater
    • California
      • San Diego, California, Forente stater, 92103
        • University of California San Diego Medical Center
      • San Francisco, California, Forente stater, 94115
        • California Pacific Medical Center
    • Florida
      • Tampa, Florida, Forente stater, 33612
        • University of South Florida
    • Georgia
      • Atlanta, Georgia, Forente stater, 30322
        • The Emory Clinic
    • Maryland
      • Baltimore, Maryland, Forente stater, 21287
        • Johns Hopkins Hospital
    • Massachusetts
      • Charlestown, Massachusetts, Forente stater, 2129
        • Massachusetts General Hospital, MA
    • Missouri
      • Saint Louis, Missouri, Forente stater, 63110
        • Washington University School of Medicine
    • Pennsylvania
      • Hershey, Pennsylvania, Forente stater, EC037
        • Penn State Milton S. Hershey Medical Center
  • Frankrike
    • Hérault
      • Montpellier, Hérault, Frankrike, 34295
        • Hopital Gui de Chauliac, Service de Neurologie
    • Paris
      • Paris cedex 13, Paris, Frankrike, 75013
        • Groupe Hospitalier Pitie-Salpetriere
  • Irland
      • Dublin, Irland, Dublin 9
        • Beaumont Hospital
  • Nederland
    • CX
      • Utrecht, CX, Nederland, 3584
        • UMC Utrecht
  • Storbritannia
    • West Midlands
      • Sheffield, West Midlands, Storbritannia, S102JF
        • Royal Hallamshire Hospital
  • Sveits
      • St. Gallen, Sveits, 9007
        • Kantonsspital St. Gallen
  • Tyskland
      • Berlin, Tyskland, 13125
        • Charite - Campus Virchow-Klinikum
      • Hannover, Tyskland, 30625
        • Medizinische Hochschule Hannover
      • Jena, Tyskland, 07743
        • Universitaetsklinikum Jena
      • Ulm, Tyskland, 89081
        • Universitaetsklinikum Ulm

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

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

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Prøvetakingsmetode

Sannsynlighetsprøve

Studiepopulasjon

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Biogen

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

6. januar 2016

Primær fullføring (Faktiske)

27. juli 2018

Studiet fullført (Faktiske)

1. august 2019

Datoer for studieregistrering

Først innsendt

8. oktober 2015

Først innsendt som oppfylte QC-kriteriene

19. november 2015

Først lagt ut (Anslag)

23. november 2015

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

24. oktober 2019

Siste oppdatering sendt inn som oppfylte QC-kriteriene

23. oktober 2019

Sist bekreftet

1. oktober 2019

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 999AS003

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