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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03160898
Ethics application status
Date submitted
12/05/2017
Date registered
17/05/2017
Date last updated
1/10/2018

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 - CK-2127107
Treatment: Drugs - Placebo

Experimental: CK-2127107 300 mg - Participants will receive CK-2127107 300 mg for 12 weeks

Experimental: CK-2127107 600 mg - Participants will receive CK-2127107 600 mg for 12 weeks

Experimental: CK-2127107 900 mg - Participants will receive CK-2127107 900 mg for 12 weeks

Placebo Comparator: Placebo - Participants will receive placebo for 12 weeks


Treatment: Drugs: CK-2127107
Oral

Treatment: Drugs: Placebo
Oral
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
Slope of change from baseline in the mega-score of muscle strength measured by hand held dynamometry and handgrip dynamometry from baseline to Week 12
Timepoint [1] 0 0
Baseline to Week 12
Secondary outcome [2] 0 0
Change from baseline to Week 12 in the ALS Functional Rating Scale - Revised (ALSFRS-R)
Timepoint [2] 0 0
Baseline to Week 12
Secondary outcome [3] 0 0
Mean plasma concentrations over time of CK-2127107 at Week 12
Timepoint [3] 0 0
Day 1 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.
Minimum age
18 Years
Maximum age
80 Years
Gender
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
Recruiting
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
1/03/2019
Actual
Sample size
Target
445
Accrual to date
Final
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
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Connecticut
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United States of America
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District of Columbia
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United States of America
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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
The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory
function and other measures of skeletal muscle function in patients with ALS.
Trial website
https://clinicaltrials.gov/show/NCT03160898
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director MD
Address 0 0
Cytokinetics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
MD Cytokinetics
Address 0 0
Country 0 0
Phone 0 0
650-624-2929
Fax 0 0
Email 0 0
medicalaffairs@cytokinetics.com
Contact person for scientific queries

No data has been provided for results reporting
Summary results
Not applicable