Dilated CardiomyopathyPhase 1NCT03062956
A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491
A Phase 1 study of Dilated Cardiomyopathy, run by MyoKardia, Inc. at 1 site in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Dilated Cardiomyopathy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 1 siteAustralia
- Study dates
- 16 Jan 2017to 28 Nov 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomized, Placebo-Controlled Study of Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-491 in Healthy Adult Volunteers
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.
Read the detailed description
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. After the 28 day screening period, the eligible subject will be admitted to the clinical site and will receive a single dose of study drug or placebo. Subjects will be confined to the clinical site for five days (Day -1 to Day 4) and will return to the clinic on Day 7 for a safety follow-up.
- Conditions
- Dilated Cardiomyopathy
- Being tested
- MYK-491 or placebo
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MYK-491 or placeboDrug
Oral suspension
| Study group | Role | What they receive |
|---|---|---|
| Single oral dose of MYK-491 | Experimental | single-dose, oral suspension Receives: Drug: MYK-491 or placebo |
| Single oral dose of placebo | Placebo comparator | single-dose, oral suspension Receives: Drug: MYK-491 or placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 7 days
Secondary outcomes (5)
Maximum observed plasma drug concentration (Cmax)
Measured: 7 days
Maximum observed plasma concentration (Tmax)
Measured: 7 days
Area under the plasma concentration-time curve (AUC)
Measured: 7 days
First-order terminal elimination half-life (t1/2)
Measured: 7 days
Mean retention time (MRT)
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2017
- Study start16 Jan 2017
- Primary completion28 Nov 2017
- Study completion28 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- MyoKardia, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jason Lickliter, MD, Principal investigator, Nucleus Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03062956
- Sponsor's protocol ID
- MYK-491-001
- Status last verified
- Mar 2018
- First submitted
- 16 Feb 2017
- Last updated
- 23 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Placebo-Controlled Study of Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-491 in Healthy Adult Volunteers
- ClinicalTrials.gov ID
- NCT03062956
- Acronym / sponsor ID
- MYK-491-001
- Conditions
- Dilated Cardiomyopathy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- MyoKardia, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Jan 2017 (actual)
- Primary completion
- 28 Nov 2017 (actual)
- Study completion
- 28 Nov 2017 (actual)
- First posted
- 24 Feb 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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. After the 28 day screening period, the eligible subject will be admitted to the clinical site and will receive a single dose of study drug or placebo. Subjects will be confined to the clinical site for five days (Day -1 to Day 4) and will return to the clinic on Day 7 for a safety follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Single oral dose of MYK-491 | Experimental | Drug: MYK-491 or placebosingle-dose, oral suspension |
| Single oral dose of placebo | Placebo comparator | Drug: MYK-491 or placebosingle-dose, oral suspension |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MYK-491 or placeboOral suspension | Drug | Single oral dose of MYK-491, Single oral dose of placebo |
Primary outcome
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
Time frame: 7 days
5 secondary outcomes
Maximum observed plasma drug concentration (Cmax)
Time frame: 7 days
Maximum observed plasma concentration (Tmax)
Time frame: 7 days
Area under the plasma concentration-time curve (AUC)
Time frame: 7 days
First-order terminal elimination half-life (t1/2)
Time frame: 7 days
Mean retention time (MRT)
Time frame: 7 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jason Lickliter, MD principal investigator
Nucleus Network
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study of Dilated Cardiomyopathy, run by MyoKardia, Inc. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 7 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2018.