Clinical trial NCT02611674: Methodology Study of Novel Outcome Measures to Assess Progression of ALS
Official title: Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2015-11-23
Enrollment: 138
Sex: ALL
Minimum age (years): 16
Maximum age (years): 85
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants suffering from ALS are recruited by participating physicians in a standard clinical practice setting.

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM)
timeFrame: Baseline to Month 6 and Baseline to Month 12
measure: Longitudinal standardized mean change in electrophysiological measures as assessed by compound muscle action potential (CMAP)
timeFrame: Baseline to Month 6 and Baseline to Month 12
measure: Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number estimation (MUNE)
timeFrame: Baseline to Month 6 and Baseline to Month 12
measure: Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number index (MUNIX)
timeFrame: Baseline to Month 6 and Baseline to Month 12
measure: Longitudinal standardized mean change in muscle strength measures as assessed by hand-held dynamometry (HHD)
timeFrame: Baseline to Month 6 and Baseline to Month 12
measure: Longitudinal standardized mean change in respiratory measures as assessed by slow vital capacity (SVC)
timeFrame: Baseline to Month 6 and Baseline to Month 12
measure: Longitudinal standardized mean change in functional measures as assessed by Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Baseline to Month 6 and Baseline to Month 12
Secondary outcomes:
measure: Within-participant test-retest reliability between the 2 repeated measurements occurring on Day 1 and Day 7 for EIM
timeFrame: Day 1 and Day 7
measure: Within-participant test-retest reliability between the 2 repeated measurements for CMAP
timeFrame: Day 1 and Day 7
measure: Within-participant test-retest reliability between the 2 repeated measurements for MUNE
timeFrame: Day 1 and Day 7
measure: Within-participant test-retest reliability between the 2 repeated measurements for MUNIX
timeFrame: Day 1 and Day 7
measure: Within-participant test-retest reliability between the 2 repeated measurements for HHD
timeFrame: Day 1 and Day 7
measure: Within-participant test-retest reliability between the 2 repeated measurements for SVC
timeFrame: Day 1 and Day 7
measure: Within-participant test-retest reliability between the 2 repeated measurements for ALSFRS-R
timeFrame: Day 1 and Day 7
measure: Comparison between 6 and 12-month changes in exploratory measures with 18 and 24-month changes in ALSFRS-R and survival
timeFrame: Baseline to Month 24
measure: Comparison between 6-month changes for muscle electrophysiological measures
timeFrame: Baseline to Month 12
measure: Comparison between 6-month changes for muscle strength measures
timeFrame: Baseline to Month 12
measure: Comparison between 6-month changes for functional measures
timeFrame: Baseline to Month 12
measure: Comparison of molecular biomarkers with disease progression
timeFrame: Baseline to Month 12
Other outcomes:
Locations:
city: San Diego
country: United States
facility: University of California San Diego Medical Center
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: The Emory Clinic
state: Georgia
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Charlestown
country: United States
facility: Massachusetts General Hospital, MA
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Leuven
country: Belgium
facility: UZ Leuven
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
state: Ontario
city: Montréal
country: Canada
facility: Montreal Neurological Institute Clinical Research Unit
state: Quebec
city: Montpellier
country: France
facility: Hopital Gui de Chauliac, Service de Neurologie
state: Hérault
city: Paris cedex 13
country: France
facility: Groupe Hospitalier Pitie-Salpetriere
state: Paris
city: Berlin
country: Germany
facility: Charite - Campus Virchow-Klinikum
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Jena
country: Germany
facility: Universitaetsklinikum Jena
city: Ulm
country: Germany
facility: Universitaetsklinikum Ulm
city: Dublin
country: Ireland
facility: Beaumont Hospital
city: Utrecht
country: Netherlands
facility: UMC Utrecht
state: CX
city: St. Gallen
country: Switzerland
facility: Kantonsspital St. Gallen
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
state: West Midlands
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: