A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Intervention: Reldesemtiv; Placebo
Intervention description: Oral tablet; Oral tablet
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 458.0
Sex: All
Minimum age: 18 Years
Maximum age: 80 Years
Healthy volunteers: No
Keywords: Amyotrophic Lateral Sclerosis; ALS; CK-2127107; Reldesemtiv

Summary:

      The purpose of this study was to assess the effect of CK-2127107 (hereafter referred to as
      reldesemtiv) versus placebo on respiratory function and other measures of skeletal muscle
      function in patients with ALS.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening

          -  Upright Slow Vital Capacity (SVC) ≥ 60% of predicted for age, height and sex at
             screening

          -  Able to swallow tablets

          -  A caregiver (if one is needed)

          -  Able to perform reproducible pulmonary function tests

          -  Pre-study clinical laboratory findings within the normal range or, if outside the
             normal range, deemed not clinically significant by the Investigator

          -  Male patients who have not had a vasectomy and confirmed zero sperm count must agree
             after receiving the first dose of study drug until 10 weeks after the last dose to
             either use acceptable methods of contraception or abstain from sex

          -  Female patients must be post-menopausal or sterilized or must not be breastfeeding,
             have a negative pregnancy test, have no intention to become pregnant during the study
             and use acceptable methods of contraception or abstain from heterosexual intercourse
             from Screening until 10 weeks after last dose of study drug

          -  Patients must be either on riluzole for at least 30 days prior to screening or have
             not taken riluzole for at least 30 days prior to screening and not planning to start
             riluzole during the course of the study.

          -  Patients on edaravone must have completed at least 2 cycles of dosing with edaravone
             at the time of screening or have not taken edaravone for at least 30 days prior to
             screening and not planning to start edaravone during the course of the study.

        Exclusion Criteria:

          -  At the time of screening, any use of non-invasive ventilation (NIV), e.g. continuous
             positive airway pressure [CPAP], noninvasive bi-level positive airway pressure [NPPV]
             or noninvasive volume ventilation [NVV] for any portion of the day, or mechanical
             ventilation via tracheostomy, or on any form of oxygen supplementation

          -  Neurological impairment due to a condition other than ALS

          -  Presence at screening of any medically significant cardiac, pulmonary, GI,
             musculoskeletal, or psychiatric illness that might interfere with the patient's
             ability to comply with study procedures or that might confound the interpretation of
             clinical safety or efficacy data

          -  Has taken any investigational study drug within 30 days or five half-lives of the
             prior agent, whichever is longer, prior to dosing

          -  Known to have received CK-2127107 or tirasemtiv in any previous clinical trial

          -  Has received or is considering receiving during the course of the study any form of
             stem cell therapy for the treatment of ALS

          -  Has received or is considering receiving during the course of the study any form of
             gene therapy for the treatment of ALS

          -  Has received or is considering obtaining during the course of the study a
             diaphragmatic pacing system

          -  History of substance abuse within the past 2 years

          -  Use of certain medications