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Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):259-266. doi: 10.1080/21678421.2018.1426770. Epub 2018 Feb 6.

VITALITY-ALS, a phase III trial of tirasemtiv, a selective fast skeletal muscle troponin activator, as a potential treatment for patients with amyotrophic lateral sclerosis: study design and baseline characteristics.

Author information

1
a Eleanor and Lou Gehrig ALS Center, Columbia University , New York , NY , USA.
2
b Department of Neurology , Massachussetts General Hospital , Boston , MA , USA.
3
c Biomedical Sciences Institute of Neurosciences, The University of Dublin , Dublin , Ireland.
4
d Cytokinetics, Inc , South San Francisco , CA , USA , and.
5
e Department of Neurology , Barrow Neurological Institute , Phoenix , AZ , USA.

Erratum in

  • Erratum. [Amyotroph Lateral Scler Frontotemporal Degener. 2018]

Abstract

OBJECTIVE:

To assess the efficacy of tirasemtiv, a fast skeletal muscle troponin activator, vs. placebo on respiratory function and other functional measures in patients with amyotrophic lateral sclerosis (ALS). This study was designed to confirm and extend results from a large phase IIb trial and maximize tolerability with a slower dose escalation.

METHODS:

VITALITY-ALS (NCT02496767) was a multinational, double-blind, randomized, placebo-controlled, parallel-group study in ALS patients. Participants who tolerated two weeks of open-label tirasemtiv (125 mg twice a day) were randomized 3:2:2:2 to placebo or one of three target total daily dose levels of tirasemtiv (250, 375, or 500 mg). Participants randomized to tirasemtiv escalated their dose every two weeks to their target dose level or maximum tolerated dose. The primary outcome measure was change in slow vital capacity from baseline to 24 weeks. Secondary endpoints assessed the effect of tirasemtiv on muscle strength and certain respiratory milestones of disease progression. A four-week randomized withdrawal phase followed 48 weeks of treatment to evaluate the possibility of sustained benefit or rebound decline.

RESULTS:

Data collection will be complete in the fourth quarter of 2017.

CONCLUSIONS:

VITALITY-ALS was a phase III trial designed to evaluate the efficacy, safety, and tolerability of tirasemtiv in ALS.

KEYWORDS:

Tirasemtiv; amyotrophic lateral sclerosis; slow vital capacity

PMID:
29402141
DOI:
10.1080/21678421.2018.1426770
[Indexed for MEDLINE]

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