title: Outcome measures in amyotrophic lateral sclerosis clinical trials.
abstract: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with an average survival of 3-5 years. While therapies for ALS remain limited, basic and translational ALS research has been host to numerous influential discoveries in recent years. These discoveries have led to a large pipeline of potential therapies that await testing in clinical trials. Until recently, ALS clinical trials have relied on a limited cadre of 'traditional' outcome measures, including survival and measures of function. These measures have proven useful, although imperfect, in Phase III ALS trials. However, their utility in early-phase ALS trials is limited. For these early trials, outcome measures focused on target engagement or biological pathway analysis might improve trial outcomes and better support the drug development process.
authors.affiliation: Harvard Medical School, Department of Neurology, Neurological Clinical Research Institute (NCRI), Massachusetts Genera Hospital, MA, USA; Harvard Medical School, Department of Physical Medicine & Rehabilitation, Spaulding Rehabilitation Hospital, Boston, MA, USA.
authors.forename: Sabrina
authors.initials: S
authors.lastname: Paganoni
authors.affiliation: Harvard Medical School, Department of Neurology, Neurological Clinical Research Institute (NCRI), Massachusetts Genera Hospital, MA, USA.
authors.forename: Merit
authors.initials: M
authors.lastname: Cudkowicz
authors.affiliation: Harvard Medical School, Department of Neurology, Neurological Clinical Research Institute (NCRI), Massachusetts Genera Hospital, MA, USA.
authors.forename: James D
authors.initials: JD
authors.lastname: Berry
keywords: biomarker; end point; function; surrogate; survival