A Phase 1, Blinded, Randomized, Placebo-controlled, Nested Ascending Single Oral Dose and Ascending Multiple Oral Dose Study (with Food Study) to Evaluate Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of MYK-224 in Healthy Volunteers

MyoKardia, Inc.0 sites112 target enrollmentApril 16, 2021

ConditionsHypertrophic Cardiomyopathy Cardiovascular - Other cardiovascular diseases

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Hypertrophic Cardiomyopathy
Sponsor: MyoKardia, Inc.
Enrollment: 112
Status: Recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

April 16, 2021

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional

Sex

All

Investigators

Sponsor

MyoKardia, Inc.

Eligibility Criteria

Inclusion Criteria

•1\. Participant is able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to federal, local, and institutional guidelines before the first study\-specific procedure.
•2\. Participant is between the ages of 18 and 45 years, inclusive, at the screening visit.
•3\. Participant has a weight greater than or equal to 50 kg and body mass index between 18 kg/m2 and 32 kg/m2, inclusive, at the screening visit, calculated via the institution’s standard formula.
•4\. Participant is healthy as determined by medical history, physical examination, vital signs, and routine laboratory parameters (chemistry, hematology, and urinalysis) at the screening visit and on day \-1\. Laboratory values outside the normal range are acceptable if: (1\) deemed to be clinically insignificant by the investigator and (2\) if transaminases (aspartate aminotransferase \[AST] or alanine aminotransferase \[ALT]), less than 1\.5 times the upper limit of the laboratory reference range.
•5\. Participant has documented LVEF greater than or equal to 55% during screening.
•6\. Participant has adequate acoustic windows, in the judgment of the core echo laboratory, to enable accurate transthoracic echocardiography (TTE) measurement of PD endpoints.
•7\. Participant has QT interval with Fridericia Correction (QTcF) less than 450 msec.
•8\. Participant has either a normal 12\-lead ECG at Screening, including standard intervals, or one with abnormalities that are considered clinically insignificant by the investigator in consultation with MyoKardia.

Exclusion Criteria

•1\. Hypersensitivity to any of the components of the MYK\-224 formulation or anaphylaxis to any drug, food, animal sting or other substance.
•2\. Any structural abnormalities on screening TTE, in the judgment of the core cardiac laboratory.
•3\. Participant has a history of clinically significant arrhythmia (including, but not limited to, any predominant cardiac rhythm other than normal sinus \[including resting heart rate \[HR] persistently greater than 100 bpm or less than 50 bpm \[repeat measurements permitted to establish or exclude and HR less than 50 and greater than 45 may be included after consultation with the medical monitor] ) the presence of clinically relevant ventricular ectopy \[frequent premature ventricular complexes, ventricular bigeminy, couplets or nonsustained ventricular tachycardia], or evidence of clinically important conduction abnormalities), LV systolic dysfunction, or coronary artery disease.
•4\. Participant has a history of malignancy of any type, other than in situ cervical cancer more than 10 years prior to screening or surgically excised nonmelanoma skin cancers more than 2 years prior to Screening.
•5\. Participant has a positive serologic test at Screening for infection with human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV).
•6\. Participant has an active infection indicated clinically as determined by the Investigator.
•7\. Participants has a positive test for alcohol or drugs of abuse at Screening or Day \-1\.
•8\. Participant has used prescription medication within 28 days of Day 1 or over the\- counter\- medication (including herbal preparations and supplements) within 14 days of Day 1 except for the following exceptions: contraceptive medication is allowed; acetaminophen/paracetamol up to 2 g per day is allowed; and prescription medication may be allowed in this window with Investigator and Sponsor approval, as long as more than 5 half\-lives have elapsed since last taking the medication before randomization and the medication is not expected to impact the safety of participants or study procedures.
•9\. Participant has used or ingested alcohol, tobacco and nicotine\-containing substances, grapefruit and grapefruit juice, Seville oranges, and quinine (eg, tonic water) within 7 days prior to check\-in on Day \-1\.Seville oranges, and quinine (eg, tonic water) within 7 days prior to check\-in on Day \-1\.