Methodology Study of Novel Outcome Measures to Assess Progression of ALS

NCT ID: NCT02611674

Last Updated: 2019-10-24

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 138 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2016-01-06
• Study Completion Date: 2019-08-01

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

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

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

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Biogen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Biogen

Locations

University of California San Diego Medical Center

San Diego, California, United States

California Pacific Medical Center

San Francisco, California, United States

University of South Florida

Tampa, Florida, United States

The Emory Clinic

Atlanta, Georgia, United States

Johns Hopkins Hospital

Baltimore, Maryland, United States

Massachusetts General Hospital, MA

Charlestown, Massachusetts, United States

Washington University School of Medicine

St Louis, Missouri, United States

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

UZ Leuven

Leuven, , Belgium

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Montreal Neurological Institute Clinical Research Unit

Montreal, Quebec, Canada

Hopital Gui de Chauliac, Service de Neurologie

Montpellier, Hérault, France

Groupe Hospitalier Pitie-Salpetriere

Paris, Paris, France

Charite - Campus Virchow-Klinikum

Berlin, , Germany

Medizinische Hochschule Hannover

Hanover, , Germany

Universitaetsklinikum Jena

Jena, , Germany

Universitaetsklinikum Ulm

Ulm, , Germany

Beaumont Hospital

Dublin, , Ireland

UMC Utrecht

Utrecht, CX, Netherlands

Kantonsspital St. Gallen

Sankt Gallen, , Switzerland

Royal Hallamshire Hospital

Sheffield, West Midlands, United Kingdom

Countries

United States; Belgium; Canada; France; Germany; Ireland; Netherlands; Switzerland; United Kingdom

Other Identifiers

999AS003

Identifier Type: -

Identifier Source: org_study_id