Phase II study of sacubitril/valsartan in patients with nHCM
Research type
Research Study
Full title
A multi-center, randomized, placebo-controlled patient and investigator-blinded study to explore the efficacy of oral sacubitril/valsartan in adult patients with non-obstructive hypertrophic cardiomyopathy (nHCM)
IRAS ID
275969
Contact name
Perry Elliott
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2019-003098-24
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 4 months, 30 days
Research summary
The purpose of this study is to determine if LCZ696 (also known as Entresto® or sacubitril/valsartan) is safe, tolerable and can improve exercise capacity in non-obstructive hypertrophic cardiomyopathy (nHCM) patients. This study will also explore changes in cardiac structure and function following treatment with LCZ696 in nHCM patients.
HCM is a genetic disorder of heart muscle cells characterised by cardiac hypertrophy (abnormal enlargement, or thickening, of the heart muscle) and a non-dilated left ventricle with preserved or increased ejection fraction (how much blood the left ventricle pumps out with each contraction). Drug treatments of patients with HCM are currently limited to those intended to improve blood flow to the heart muscle and symptoms. Septal reduction therapy, (i.e. reduction of the heart muscle by surgery or alcohol) is an invasive treatment in obstructive HCM patients who are symptomatic and not responding to drug treatment. Advanced stages of heart failure who have failed drug therapy and, when indicated, septal reduction therapy, may require implantation of a left ventricular device or heart transplantation. Therefore, there is still an unmet medical need for new therapies for HCM.
Clinically, several LCZ696 studies have been completed to date, showing clear benefits to patients with cardiovascular disease, including heart failure and hypertension, in which vasoconstriction, volume expansion, and target organ damage (i.e. fibrosis, hypertrophy, myocardial/vascular stiffness) play a key role in their pathophysiology.
This study will enrol adult patients with nHCM and will be conducted in hospitals and clinics in countries worldwide. The study includes the following stages: screening/baseline; single-blind (i.e. patient-blinded) treatment run-in; randomised, placebo-controlled, double-blind (patient and investigator-blinded); and end of study visit. The treatment period will last 50 weeks and the maximum study duration is approximately 58 weeks. Assessments will include: blood samples, cardiopulmonary exercise testing (CPET), ECGs, echocardiograms, questionnaires, physical exams and vitals.REC name
North West - Haydock Research Ethics Committee
REC reference
20/NW/0161
Date of REC Opinion
30 Apr 2020
REC opinion
Further Information Favourable Opinion