A PHASE 3, MULTI-CENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED TRIAL TO EVALUATE THE EFFICACY AND SAFETY OF RELDESEMTIV IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS (ALS)

Phase 3

Completed

Conditions: ALS
Amyotrophic Lateral Sclerosis
10029317

Registration Number: NL-OMON52262

Lead Sponsor: Cytokinetics Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 10

Inclusion Criteria

Males or Females between the ages of 18 and 80 years of age, inclusive

• Diagnosis of familial or sporadic ALS (defined as meeting the
laboratory-supported probable, probable, or definite criteria for ALS according
to the World Federation of Neurology El Escorial criteria published in 2000
[Brooks 2000]). Patients who meet the possible criteria are eligible if they
have lower motor neuron findings; those who have purely upper motor neuron
findings are ineligible.

• First symptom of ALS <= 24 months prior to screening. The qualifying first
symptoms of ALS are limited to manifestations of weakness in extremity, bulbar,
or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken
in isolation as a first symptom of ALS.
• ALSFRS-R total score <= 44 at screening. Patients with a total score of 45 or
higher may be rescreened 60±7 days following the original screening date and be
deemed eligible if their ALSFRS-R total score is <= 44 or if their score is 2 or
more points less than at initial screening.
Such patients must continue to meet all other inclusion/exclusion criteria at
the time of rescreening.
• Upright FVC >= 65.0% of predicted for age, height, sex and ethnicity at
screening according to
Global Lung Initiative equation
• Able to perform reproducible pulmonary function tests defined as being able
to perform FVC at screening with variability of the 2 highest raw values of
less than 10% with a maximum of 5 trials permitted. Screening FVC results must
be reviewed and approved by the central review process prior to randomization.
• Must be either on riluzole for >= 30 days prior to screening or have not taken
it for at least 30 days
prior to screening
• Must have completed at least 2 cycles of edaravone at the time of screening
or have not received it for at least 30 days prior to screening
• Able to swallow whole tablets at the time of screening
. Clinical laboratory findings within the normal range, or if outside the
normal range, not deemed clinically significant by the Investigator, except as
specifically indicated as laboratory exclusion

Exclusion Criteria

• eGFRCr and eGFRCysC < 45.0 mL/min/1.73 m2 at screening

• Urine protein/creatinine ratio > 1 mg/mg (113 mg/mmol) at screening

• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >= 3-times
the upper limit of normal (ULN)

• Total bilirubin (TBL), direct or indirect bilirubin above the ULN.

• Cognitive impairment, related to ALS or otherwise that impairs the patient*s
ability to understand and/or comply with study procedures and provide informed
consent

• Other medically significant neurological conditions that could interfere with
the assessment of ALS
symptoms, signs or progression.

• Presence at screening of any medically significant cardiac, pulmonary,
gastrointestinal, 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 a tracheostomy

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary study outcome is the change from baseline to Week 24 in ALSFRS-R<br /><br>total score.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The secondary outcomes of study are:<br /><br>• Combined assessment of change in ALSFRS-R total score, time to onset of<br /><br>respiratory insufficiency, and survival time up to Week 24<br /><br><br /><br>• Change from baseline to Week 24 in the percent predicted FVC<br /><br><br /><br>• Change from baseline to Week 24 in the<br /><br>ALSAQ-40 total score<br /><br><br /><br>• Change from baseline to Week 24 in handgrip strength (average of both hands).</p><br>