Methodology Study of Novel Outcome Measures to Assess Progression of ALS

Status:	Active, not recruiting
Conditions:	Neurology, Neurology, Neurology, ALS
Therapuetic Areas:	Neurology, Other
Healthy:	No
Age Range:	16 - 85
Updated:	8/10/2018
Start Date:	January 6, 2016
End Date:	August 2, 2019

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 novel 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.

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

We found this trial at

sites

California Pacific Medical Center

45 Castro Street
San Francisco, California 94114

(415) 600-6000