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Methodology Study of Novel Outcome Measures to Assess Progression of ALS

23 oktober 2019 bijgewerkt door: 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.

Studie Overzicht

Toestand

Voltooid

Conditie

Amyotrofische laterale sclerose

Studietype

Observationeel

Inschrijving (Werkelijk)

138

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

16 jaar tot 85 jaar (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Bemonsteringsmethode

Kanssteekproef

Studie Bevolking

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

Beschrijving

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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat	Maatregel Beschrijving	Tijdsspanne
Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM) Tijdsspanne: 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) Tijdsspanne: 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) Tijdsspanne: 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) Tijdsspanne: 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) Tijdsspanne: 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) Tijdsspanne: 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) Tijdsspanne: 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

Secundaire uitkomstmaten

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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Biogen

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

6 januari 2016

Primaire voltooiing (Werkelijk)

27 juli 2018

Studie voltooiing (Werkelijk)

1 augustus 2019

Studieregistratiedata

Eerst ingediend

8 oktober 2015

Eerst ingediend dat voldeed aan de QC-criteria

19 november 2015

Eerst geplaatst (Schatting)

23 november 2015

Updates van studierecords

Laatste update geplaatst (Werkelijk)

24 oktober 2019

Laatste update ingediend die voldeed aan QC-criteria

23 oktober 2019

Laatst geverifieerd

1 oktober 2019

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

999AS003

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