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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03062956




Registration number
NCT03062956
Ethics application status
Date submitted
16/02/2017
Date registered
24/02/2017
Date last updated
23/03/2018

Titles & IDs
Public title
A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491
Scientific title
Randomized, Placebo-Controlled Study of Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-491 in Healthy Adult Volunteers
Secondary ID [1] 0 0
MYK-491-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dilated Cardiomyopathy 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - MYK-491 or placebo

Experimental: Single oral dose of MYK-491 - single-dose, oral suspension

Placebo Comparator: Single oral dose of placebo - single-dose, oral suspension


Treatment: Drugs: MYK-491 or placebo
Oral suspension
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability assessments will include treatment emergent AEs and SAEs, ECG recordings, vital signs, hs-troponin 1 concentrations, laboratory abnormalities and physical exam abnormalities
Timepoint [1] 0 0
7 days
Secondary outcome [1] 0 0
Maximum observed plasma drug concentration (Cmax)
Timepoint [1] 0 0
7 days
Secondary outcome [2] 0 0
Maximum observed plasma concentration (Tmax)
Timepoint [2] 0 0
7 days
Secondary outcome [3] 0 0
Area under the plasma concentration-time curve (AUC)
Timepoint [3] 0 0
7 days
Secondary outcome [4] 0 0
First-order terminal elimination half-life (t1/2)
Timepoint [4] 0 0
7 days
Secondary outcome [5] 0 0
Mean retention time (MRT)
Timepoint [5] 0 0
7 days

Eligibility
Key inclusion criteria
- Weight between 60 and 90 kg inclusive

- Resting heart rate of < 80 beats per minute

- Documented LVEF greater than or equal to 55% during Screening

- Normal electrocardiogram (ECG) at Screening

- Normal acoustic windows on transthoracic echocardiograms at Screening

- All safety laboratory parameters within normal limits at Screening

- History or evidence of another clinically significant disorder, in the opinion of the
investigator.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Active infection

- History of coronary artery disease

- History of malignancy with the exception of in situ cervical cancer more than 5 years
prior to Screening or surgically-excised non-melanomatous skin cancers more than 2
years prior to Screening

- Positive serology tests at screening

- Current use of tobacco or nicotine-containing products exceeding 10 per day.

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
Other
Other design features
Phase
Phase 1
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
16/01/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/11/2017
Sample size
Target
Accrual to date
Final
64
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
MyoKardia, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Up to 72 healthy volunteers will be given a single dose of MYK-491 or placebo and be
monitored for safety and tolerability over a 7 day period.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03062956
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jason Lickliter, MD
Address 0 0
Nucleus Network
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03062956