Methodology Study of Novel Outcome Measures to Assess Progression of ALS

Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis

Sponsors

Lead Sponsor: Biogen

Source Biogen
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.

Overall Status Completed
Start Date 2016-01-06
Completion Date 2019-08-01
Primary Completion Date 2018-07-27
Study Type Observational
Primary Outcome
Measure Time Frame
Longitudinal standardized mean change in electrophysiological measures as assessed by electrical impedance myography (EIM) Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in electrophysiological measures as assessed by compound muscle action potential (CMAP) Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number estimation (MUNE) Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in electrophysiological measures as assessed by motor unit number index (MUNIX) Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in muscle strength measures as assessed by hand-held dynamometry (HHD) Baseline to Month 6 and Baseline to Month 12
Longitudinal standardized mean change in respiratory measures as assessed by slow vital capacity (SVC) 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) Baseline to Month 6 and Baseline to Month 12
Secondary Outcome
Measure Time Frame
Within-participant test-retest reliability between the 2 repeated measurements occurring on Day 1 and Day 7 for EIM Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for CMAP Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for MUNE Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for MUNIX Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for HHD Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for SVC Day 1 and Day 7
Within-participant test-retest reliability between the 2 repeated measurements for ALSFRS-R 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 Baseline to Month 24
Comparison between 6-month changes for muscle electrophysiological measures Baseline to Month 12
Comparison between 6-month changes for muscle strength measures Baseline to Month 12
Comparison between 6-month changes for functional measures Baseline to Month 12
Comparison of molecular biomarkers with disease progression Baseline to Month 12
Enrollment 138
Condition
Eligibility

Sampling Method:

Probability Sample

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

Gender:

All

Minimum Age:

16 Years

Maximum Age:

85 Years

Healthy Volunteers:

No

Overall Official
Last Name Role Affiliation
Medical Director Study Director Biogen
Location
Facility:
University of California San Diego Medical Center | San Diego, California, 92103, United States
California Pacific Medical Center | San Francisco, California, 94115, United States
University of South Florida | Tampa, Florida, 33612, United States
The Emory Clinic | Atlanta, Georgia, 30322, United States
Johns Hopkins Hospital | Baltimore, Maryland, 21287, United States
Massachusetts General Hospital, MA | Charlestown, Massachusetts, 2129, United States
Washington University School of Medicine | Saint Louis, Missouri, 63110, United States
Penn State Milton S. Hershey Medical Center | Hershey, Pennsylvania, EC037, United States
UZ Leuven | Leuven, 3000, Belgium
Sunnybrook Health Sciences Centre | Toronto, Ontario, M4N 3M5, Canada
Montreal Neurological Institute Clinical Research Unit | Montréal, Quebec, H3A 2B4, Canada
Hopital Gui de Chauliac, Service de Neurologie | Montpellier, Hérault, 34295, France
Groupe Hospitalier Pitie-Salpetriere | Paris cedex 13, Paris, 75013, France
Charite - Campus Virchow-Klinikum | Berlin, 13125, Germany
Medizinische Hochschule Hannover | Hannover, 30625, Germany
Universitaetsklinikum Jena | Jena, 07743, Germany
Universitaetsklinikum Ulm | Ulm, 89081, Germany
Beaumont Hospital | Dublin, Dublin 9, Ireland
UMC Utrecht | Utrecht, CX, 3584, Netherlands
Kantonsspital St. Gallen | St. Gallen, 9007, Switzerland
Royal Hallamshire Hospital | Sheffield, West Midlands, S102JF, United Kingdom
Location Countries

Belgium

Canada

France

Germany

Ireland

Netherlands

Switzerland

United Kingdom

United States

Verification Date

2019-10-01

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

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