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Methodology Study of Novel Outcome Measures to Assess Progression of ALS

23 de octubre de 2019 actualizado por: 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.

Descripción general del estudio

Estado

Terminado

Tipo de estudio

De observación

Inscripción (Actual)

138

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

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

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

16 años a 85 años (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Método de muestreo

Muestra de probabilidad

Población de estudio

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

Descripción

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 de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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

Medidas de resultado secundarias

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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

6 de enero de 2016

Finalización primaria (Actual)

27 de julio de 2018

Finalización del estudio (Actual)

1 de agosto de 2019

Fechas de registro del estudio

Enviado por primera vez

8 de octubre de 2015

Primero enviado que cumplió con los criterios de control de calidad

19 de noviembre de 2015

Publicado por primera vez (Estimar)

23 de noviembre de 2015

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

24 de octubre de 2019

Última actualización enviada que cumplió con los criterios de control de calidad

23 de octubre de 2019

Última verificación

1 de octubre de 2019

Más información

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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