Exploratory Study of MYK-491 in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants

Título: 
Exploratory Study of MYK-491 in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants
Estado de reclutamiento: 
Estado se actualizó más recientemente: 
October 1, 2020
Gene(s): 
Propósito de estudio: 

The purpose of this Phase 2a study is to establish safety and tolerability of treatment with MYK-491 in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants.

Intervención / tratamiento: 
Drug: danicamtiv
Fase: 
Descripción del estudio: 

N/A

Tipo de estudio: 
Título oficial: 
Open-Label Exploratory Study of Oral MYK-491 in Stable Ambulatory Patients With Primary Dilated Cardiomyopathy Due to Either MYH7 or TTN Variants
Fecha de inicio del estudio: 
August 19, 2020
Fecha de finalización del estudio: 
July 2021
Objetivo(s) principal(es): 

Frequency and severity of treatment-emergent adverse events and serious adverse events. [ Time Frame: Up to 22 days ]

Objetivo(s) secundario(s): 

N/A

Elegibilidad: 

Ages Eligible for Study:  18 Years to 80 Years   (Adult, Older Adult)

Sexes Eligible for Study:  All

Accepts Healthy Volunteers:  No

Criterios de inclusión: 

Has stable primary dilated cardiomyopathy due to either MYH7 or TTN variant

Has adequate acoustic windows for echocardiography

Maximum of 3 family members with same variant can be enrolled

Criterio de exclusión: 
  • Any significant structural cardiac abnormalities on Screening transthoracic echo(s)
  • A pathogenic variant implicated in DCM of another gene other than MYH7 or TTN
  • Routinely scheduled outpatient intravenous (IV) infusions for heart failure (e.g., inotropes, vasodilators [e.g., nesiritide], diuretics), or routinely scheduled ultrafiltration
  • Presence of protocol specified laboratory abnormalities at Screening
Sitio(s) de estudio / Ubicacion(es): 
  • United States, Massachusetts
  • Brigham and Women's HospitalRecruiting
  • Boston, Massachusetts, United States, 02115
  • Contact: Mary Sheehan, RN    617-732-6237      
Patrocinadores y Colaboradores: 

MyoKardia, Inc.

Investigador(es) principal(es): 

Myokardia Medical Information

650-741-0900

medinfo@myokardia.com

Para obtener más información, por favor comuníquese con el Coordinador del estudio: 

Contact:  

Email: 

Phone: 

ID de ClinicalTrials.gov: 
NCT04572893