Study Purpose:
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.Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Observational
Type of Intervention:
N/A
Intervention Name:
N/A
Placebo:
N/A
Phase:
N/A
Study Chair(s)/Principal Investigator(s):
Medical Director, Biogen
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
United States
Full Study 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 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.
Study Sponsor:
Biogen
Estimated Enrollment:
138
Estimated Study Start Date:
01 / 06 / 2016
Estimated Study Completion Date:
08 / 01 / 2019
Posting Last Modified Date:
10 / 24 / 2019
Date Study Added to neals.org:
11 / 23 / 2015
Minimum Age:
16 Years
Maximum Age:
85 Years
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
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