AN OPEN-LABEL, EXPLORATORY STUDY OF THE SAFETY AND PRELIMINARY EFFICACY OF DANICAMTIV IN STABLE AMBULATORY PARTICIPANTS WITH PRIMARY DILATED CARDIOMYOPATHY DUE TO EITHER MYH7 OR TTN VARIANTS OR OTHER CAUSALITIES - CV028-005

Myokardia Inc.0 个研究点目标入组 24 人开始时间: 2023年7月5日最近更新: 2024年7月22日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Myokardia Inc.
入组人数: 24

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: 18 至 65+（—）
性别: All

入选标准

•Part A_I1. 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, I3_(b). Pathogenic or likely pathogenic variants submitted in the form of final official genetic laboratory reports will be reviewed centrally by the Sponsor and coordinating investigator for eligibility. Some variants designated VUS may also be permitted upon central review., I3_(c). DCM is not secondary to long-standing MYH7 or TTN-related hypertrophic cardiomyopathy (HCM) or left ventricle noncompaction cardiomyopathy, as determined by the Investigator., I3_(d). Participants with DCM related to pathogenic or likely pathogenic variants of TTN must not also have a diagnosis of peripartum DCM (DCM diagnosed initially in the last month of pregnancy or the 6 months following delivery)., I3_(e). In participants with DCM related to pathogenic or likely pathogenic variants of TTN, DCM must not be secondary to significant exposure to cardiotoxic chemotherapy agents as determined by the investigator., I3_(f). In participants with DCM related to pathogenic or likely pathogenic variants of TTN, DCM must not be due to a history of significant alcohol abuse as determined by the investigator, Part A_I7. Male participants must use barrier method of contraception (whether or not the participant had vasectomy), Part A_I8. I8. For the cohort of primary DCM due to other causalities than MYH7 and TTN, participant must meet the following criteria in addition to I1, I2, I3 (g) and (h), I4, I5, I6, and I7. a. The cause of DCM is not related to MYH7 or TTN variants b. The cause of primary DCM is by variants of the other genes except MYH7 and TTN, or non-genetic cause., I3_(g). Documented left ventricular ejection fraction (LVEF) 15-45% (on 2 occasions), including at least once during Screening and confirmed by the Echo Core Laboratory. If a participant's most recent prior TTE (within past 12 months) documents a LVEF =45%, then only a single screening visit confirming LVEF =45% by the Echo Core Laboratory is required. If no prior documented LVEF =45% by TTE within past 12 months is available, then 2 screening TTEs are needed at least one week (7 days) apart. In addition, the absolute difference between the 2 LVEF values qualifying the subject should <12%., I3_(h). Participant receives chronic medication for the treatment of heart failure reflecting current guidelines, including at least one of the following, unless not tolerated or contraindicated: ß-blocker, angiotensin converting enzyme inhibitor, angiotensin receptor blocker, or angiotensin receptor neprilysin inhibitor. Such treatments should have been given at stable doses for = 2 weeks with no plan to modify during the study, Part A_I4. Sinus rhythm or stable atrial or ventricular pacing or persistent atrial fibrillation that is adequately rate-controlled to allow PD assessments by TTE., Part A_I5. If multiple members of a family meet eligibility criteria, a maximum of 3 eligible subjects per family may enroll in the study, Part A_I6. Female of childbearing potential (Appendix 6) must not be pregnant or lactating and, if sexually active, must use one of the following highlyeffective birth control methods from the Screening visit through 3 months after the last dose of study drug: -combined (estrogen- and progestogen-containing) hormonal contracept

排除标准

•Part A_E1. Inadequate echocardiographic acoustic windows., Part A_E18. Prior/Concurrent Clinical Study Experience: Participated in a clinical trial in which the participant received any investigational drug (or is currently using an investigational device) within 30 days prior to Screening, or at least 5 times the respective elimination half-life (whichever is longer)., Part A_E19. WOCBP with a positive pregnancy test, Part A_E10. Hypersensitivity to danicamtiv or any of the components of the danicamtiv formulation, Part A_E20. Is employed by or is a first-degree relative of someone employed by the Sponsor, the investigator, or his/her staff or family, Part A_E21. Currently placed in hospital or facility due to legal or administrative order, Part A_E11. Active infection, indicated clinically as determined by the investigator. In the case of SARS-CoV-2 (COVID-19) infection within 4 weeks prior to and during Screening, symptoms must have completely resolved and based on Investigator assessment in consultation with the Clinical Trial Physician, there are no sequelae that would place the participant at a higher risk of receiving investigational treatment. The methods to assess SARS-CoV-2 (COVID-19) infection include PCR, antigen test and serology tests. Each study site should follow requirements per local institutional or regulatory guidance if any., Part A_E12. History of malignancy of any type within 5 years prior to Screening, with the exception of the following surgically excised cancers occurring more than 2 years prior to Screening: in situ cervical cancer, nonmelanomatous skin cancers, ductal carcinoma in situ, and nonmetastatic prostate cancer, Part A_E13. Severe renal insufficiency (defined as current estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73m2 by simplified Modification of Diet in Renal Disease equation [sMDRD]), Part A_E14. Serum potassium < 3.5 or > 5.5 mEq/L, Part A_E15. Any persistent (2 or more) out-of-range laboratory parameters (chemistry, hematology) at Screening, considered by the investigator and the medical monitor to be clinically significant., Part A_E4. HFrEF considered to be caused primarily by ischemic heart disease, chronic valvulopathy, or another condition, as determined by the Investigator, Part A_E2. A participant has a QTcF interval > 480 msec (not attributable to ventricular pacing or prolonged QRS duration = 120 msec, average of triplicate electrocardiograms (ECGs), Part A_E3. (a) For MYH7 and TTN cohorts, participants with known pathogenic variant of another gene implicated in DCM at screening (b) For the cohort of participants with primary DCM due to other causalities than MYH7 and TTN, known in MYH7 or TTN variants implicated in DCM at Screening., Part B_E1. Recent (< 90 days) acute coronary syndrome or angina pectoris, Part B_E2. Coronary revascularization (percutaneous coronary intervention or coronary artery bypass graft) within prior 90 days, Part B_E3. Recent (< 90 days) hospitalization for heart failure, use of intravenous diuretic or chronic intravenous inotropic therapy or other cardiovascular event (eg, cerebrovascular accident), Part B_E4. Active infection, indicated clinically as determined by the Investigator. In the case of SARS-CoV-2 (COVID-19) infection within 4 weeks prior to Part B Baseline Visit or Rescreening (if required), symptoms must have completely resolved and based on Investigator assessment in consultation with the Clinical Trial Physician, there are

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发起方

Myokardia Inc.

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