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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03160898




Registration number
NCT03160898
Ethics application status
Date submitted
12/05/2017
Date registered
19/05/2017

Titles & IDs
Public title
A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Scientific title
A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Secondary ID [1] 0 0
CY 5022
Universal Trial Number (UTN)
Trial acronym
FORTITUDE-ALS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Reldesemtiv
Treatment: Drugs - Placebo

Experimental: Reldesemtiv 150 mg twice daily - Patients in this arm took 1 reldesemtiv 150 mg oral tablet and 2 matching placebo tablets every 12 hours for 12 weeks.

Experimental: Reldesemtiv 300 mg twice daily - Patients in this arm took 2 reldesemtiv 150 mg oral tablets and 1 matching placebo tablet every 12 hours for 12 weeks.

Experimental: Reldesemtiv 450 mg twice daily - Patients in this arm took 3 reldesemtiv 150 mg oral tablets every 12 hours for 12 weeks.

Placebo comparator: Placebo - Patients in this arm took 3 placebo oral tablets every 12 hours for 12 weeks.


Treatment: Drugs: Reldesemtiv
Oral tablet

Treatment: Drugs: Placebo
Oral tablet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline to Week 12 in the Percent Predicted Slow Vital Capacity (SVC)
Timepoint [1] 0 0
Baseline to Week 12
Secondary outcome [1] 0 0
Change From Baseline to Week 12 in the ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score
Timepoint [1] 0 0
Baseline to Week 12
Secondary outcome [2] 0 0
Slope of Muscle Strength Mega-score From Baseline to Week 12
Timepoint [2] 0 0
Baseline to Week 12

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/07/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/03/2019
Sample size
Target
Accrual to date
Final
458
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA
Recruitment hospital [1] 0 0
Brain and Mind Centre, The University of Sydney - Camperdown
Recruitment hospital [2] 0 0
Department of Neurology, Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [4] 0 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [5] 0 0
The Perron Institute for Neurological and Translation Science - Nedlands
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
5042 - Bedford Park
Recruitment postcode(s) [5] 0 0
6009 - Nedlands
Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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Connecticut
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District of Columbia
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Florida
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Georgia
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Illinois
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Indiana
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Iowa
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Kansas
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Missouri
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Nebraska
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Tennessee
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Texas
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Vermont
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Virginia
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Washington
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West Virginia
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Wisconsin
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Canada
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Alberta
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Canada
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Ontario
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Canada
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Quebec
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Canada
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Saskatchewan
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Ireland
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Dublin
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Netherlands
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Utrecht
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Spain
State/province [36] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cytokinetics
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Astellas Pharma Inc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
MD Cytokinetics
Address 0 0
Cytokinetics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03160898