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Methodology Study of Novel Outcome Measures to Assess Progression of ALS

23 octobre 2019 mis à jour par: 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.

Aperçu de l'étude

Statut

Complété

Les conditions

La sclérose latérale amyotrophique

Type d'étude

Observationnel

Inscription (Réel)

138

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

16 ans à 85 ans (Enfant, Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

Méthode d'échantillonnage

Échantillon de probabilité

Population étudiée

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

La description

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

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats	Description de la mesure	Délai
Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM) Délai: 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) Délai: 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) Délai: 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) Délai: 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) Délai: 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) Délai: 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) Délai: 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

Mesures de résultats secondaires

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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Biogen

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

6 janvier 2016

Achèvement primaire (Réel)

27 juillet 2018

Achèvement de l'étude (Réel)

1 août 2019

Dates d'inscription aux études

Première soumission

8 octobre 2015

Première soumission répondant aux critères de contrôle qualité

19 novembre 2015

Première publication (Estimation)

23 novembre 2015

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

24 octobre 2019

Dernière mise à jour soumise répondant aux critères de contrôle qualité

23 octobre 2019

Dernière vérification

1 octobre 2019

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

999AS003

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