Hypertrophic CardiomyopathyPhase 1NCT02329184
Study Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-461
A Phase 1 study testing MYK-461 in people with Hypertrophic Cardiomyopathy, run by MyoKardia, Inc. at 7 sites in United States.
Trial summary
The purpose of this study is to establish initial safety, tolerability, pharmacokinetics and pharmacodynamics of MYK-461 in human subjects. This is a sequential group, single ascending (oral) dose study in NYHA Class I, II, or III patient volunteers aged 18-65 years. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hypertrophic Cardiomyopathy
- What is being tested?
- MYK-461
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 7 sitesUnited States
- Study dates
- Dec 2014to Apr 2016
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosed with HCM
- Normal left ventricular ejection fraction (LVEF)
- NYHA class I, II or III
Who cannot join
- Inherited metabolic disorders, myocardial infiltration or cardiofaciocutaneous syndrome (e.g., Noonan's, Fabry's).
- History of clinically important atrial or ventricular arrhythmias
- History of positive human immunodeficiency virus (HIV) test and/or seropositive for hepatitis C virus (HCV) or hepatitis B virus (HBV)..
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Study site
Boston, Massachusetts, 02111
Study site
Cypress, California, 90630
Study site
Durham, North Carolina, 27710
Study site
Kalamazoo, Michigan, 49007
Study site
Nashville, Tennessee, 37232
Study site
Scottsdale, Arizona, 85259
Study site
Stanford, California, 94305
About this study
Official title: Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-461 in Patient Volunteers With Hypertrophic Cardiomyopathy
The purpose of this study is to establish initial safety, tolerability, pharmacokinetics and pharmacodynamics of MYK-461 in human subjects. This is a sequential group, single ascending (oral) dose study in NYHA Class I, II, or III patient volunteers aged 18-65 years.
- Conditions
- Hypertrophic Cardiomyopathy
- Being tested
- MYK-461
- Medical subject headings
- Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MYK-461Drug
| Study group | Role | What they receive |
|---|---|---|
| MYK-461 | Experimental | Not described Receives: Drug: MYK-461 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of adverse events
Measured: 28 Days
Secondary outcomes (4)
Determination of pharmacokinetics parameters as measured by Cmax
maximum concentration (Cmax)
Measured: 28 Days
Determination of pharmacokinetics parameters as measured by Tmax
time of the maximum measured concentration (Tmax)
Measured: 28 Days
Determination of pharmacokinetics parameters as measured by AUC
area under the concentration-time curve (AUC)
Measured: 28 Days
Determination of pharmacokinetics parameters as measured by t1/2
half-life (t1/2)
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2014 (estimated)
- Study startDec 2014
- Primary completionMar 2016
- Study completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypertrophic 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- MyoKardia, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jonathan C Fox, MD, PhD, Study director, MyoKardia, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02329184
- Sponsor's protocol ID
- MYK-461-001
- Status last verified
- Sep 2016
- First submitted
- 19 Dec 2014
- Last updated
- 14 Sep 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-461 in Patient Volunteers With Hypertrophic Cardiomyopathy
- ClinicalTrials.gov ID
- NCT02329184
- Acronym / sponsor ID
- MYK-461-001
- Conditions
- Hypertrophic Cardiomyopathy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- MyoKardia, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- Mar 2016 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 31 Dec 2014 (estimated)
- 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.
The purpose of this study is to establish initial safety, tolerability, pharmacokinetics and pharmacodynamics of MYK-461 in human subjects. This is a sequential group, single ascending (oral) dose study in NYHA Class I, II, or III patient volunteers aged 18-65 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MYK-461 | Experimental | Drug: MYK-461 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosed with HCM
- Normal left ventricular ejection fraction (LVEF)
- NYHA class I, II or III
Key exclusion criteria (3 listed)
- Inherited metabolic disorders, myocardial infiltration or cardiofaciocutaneous syndrome (e.g., Noonan's, Fabry's).
- History of clinically important atrial or ventricular arrhythmias
- History of positive human immunodeficiency virus (HIV) test and/or seropositive for hepatitis C virus (HCV) or hepatitis B virus (HBV)..
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-461 | Drug | MYK-461 |
Primary outcome
Safety: Incidence of adverse events
Time frame: 28 Days
4 secondary outcomes
Determination of pharmacokinetics parameters as measured by Cmax
Time frame: 28 Days
Determination of pharmacokinetics parameters as measured by Tmax
Time frame: 28 Days
Determination of pharmacokinetics parameters as measured by AUC
Time frame: 28 Days
Determination of pharmacokinetics parameters as measured by t1/2
Time frame: 28 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 14 Sep 2016.
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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.
Jonathan C Fox, MD, PhD study director
MyoKardia, Inc.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MYK-461 in people with Hypertrophic Cardiomyopathy, run by MyoKardia, Inc. at 7 sites in United States.
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 3 inclusion and 3 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 14 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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. 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: Incidence of adverse events (time frame: 28 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 14 Sep 2016.