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Study of Efficacy of Oral Sacubitril/Valsartan in Adult Patients With Non-obstructive Hypertrophic Cardiomyopathy

Primary Purpose

Cardiomyopathy, Hypertrophic

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LCZ696
Placebo
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cardiomyopathy, Hypertrophic focused on measuring non-obstructive, hypertrophic cardiomyopathy, genetic cardiomyopathy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Diagnosed with Hypertrophic Cardiomyopathy with a left ventricular wall thickness greater than or equal to 13mm as determined by the echocardiogram obtained during the screening/baseline period
  • Left ventricular ejection fraction (LVEF) greater than or equal to 50% as determined by echocardiogram obtained during the screening/baseline period

  • Symptoms consistent with New York Heart Association (NYHA) Class II-III heart failure by physician assessment, or asymptomatic/NYHA Class I patients with:

    • NT-proBNP blood sample levels above 250 pg/ml and
    • peak VO2 of less than or equal to 80% of predicted based on age and gender as determined by cardiopulmonary exercise testing

Exclusion Criteria:

  • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for ≥7 days after stopping study drug
  • Patients with a resting or provokable left ventricular outflow tract gradient of greater than or equal to 30mm Hg
  • Septal reduction procedure within 3 months of the screening/baseline visit
  • History of atrial fibrillation within 6 months of the screening/baseline visit or placement of ICD for secondary prevention
  • Patients with a peak VO2 on the screening/baseline cardiopulmonary exercise test of > 80% of predicted based on age and gender
  • Patients who require treatment with ACE inhibitors, angiotensin receptor blockers (ARBs), or renin inhibitors
  • Known infiltrative or storage disorder such as Fabry disease, or amyloidosis
  • Known or suspected symptomatic coronary artery diseases or evidence of prior myocardial infarction
  • Systolic blood pressure of <100 mmHg or symptomatic hypotension during the screening/baseline period or treatment run-in period
  • Contraindication to ARB administration or prior history of angioedema
  • Persistent uncontrolled hypertension

Other protocol-defined inclusion/exclusion criteria may apply

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

LCZ696 at doses of 50mg, 100mg and 200mg b.i.d

Placebo to LCZ696

Arm Description

randomized in a 1:1 ratio: LCZ696 to placebo

randomized in a 1:1 ratio: LCZ696 to placebo

Outcomes

Primary Outcome Measures

Change from baseline in peak VO2 as measured by cardiopulmonary exercise test (CPET)
The primary analysis will assess the effect of LCZ696 on the change from baseline in peak VO2 (ml/kg/min) at week 50 compared to placebo, where baseline peak VO2 will come from the screening/baseline CPET.

Secondary Outcome Measures

Full Information

First Posted
November 13, 2019
Last Updated
September 27, 2023
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04164732
Brief Title
Study of Efficacy of Oral Sacubitril/Valsartan in Adult Patients With Non-obstructive Hypertrophic Cardiomyopathy
Official 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)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
January 8, 2020 (Actual)
Primary Completion Date
August 22, 2023 (Actual)
Study Completion Date
August 22, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to determine if LCZ696 is safe, tolerable and can improve exercise capacity (via improved peak VO2) in non-obstructive HCM patient population over the course of 50 weeks of treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cardiomyopathy, Hypertrophic
Keywords
non-obstructive, hypertrophic cardiomyopathy, genetic cardiomyopathy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Masking Description
Investigator and subject will be blinded to treatment allocation during the treatment period
Allocation
Randomized
Enrollment
45 (Actual)

8. Arms, Groups, and Interventions

Arm Title
LCZ696 at doses of 50mg, 100mg and 200mg b.i.d
Arm Type
Experimental
Arm Description
randomized in a 1:1 ratio: LCZ696 to placebo
Arm Title
Placebo to LCZ696
Arm Type
Placebo Comparator
Arm Description
randomized in a 1:1 ratio: LCZ696 to placebo
Intervention Type
Drug
Intervention Name(s)
LCZ696
Other Intervention Name(s)
sacubitril, valsartan
Intervention Description
LCZ696 at doses of 50mg, 100mg and 200mg b.i.d oral
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
placebo
Primary Outcome Measure Information:
Title
Change from baseline in peak VO2 as measured by cardiopulmonary exercise test (CPET)
Description
The primary analysis will assess the effect of LCZ696 on the change from baseline in peak VO2 (ml/kg/min) at week 50 compared to placebo, where baseline peak VO2 will come from the screening/baseline CPET.
Time Frame
Baseline to 50 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosed with Hypertrophic Cardiomyopathy with a left ventricular wall thickness greater than or equal to 13mm as determined by the echocardiogram obtained during the screening/baseline period Left ventricular ejection fraction (LVEF) greater than or equal to 50% as determined by echocardiogram obtained during the screening/baseline period Symptoms consistent with New York Heart Association (NYHA) Class II-III heart failure by physician assessment, or asymptomatic/NYHA Class I patients with: NT-proBNP blood sample levels above 250 pg/ml and peak VO2 of less than or equal to 80% of predicted based on age and gender as determined by cardiopulmonary exercise testing Exclusion Criteria: Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for ≥7 days after stopping study drug Patients with a resting or provokable left ventricular outflow tract gradient of greater than or equal to 30mm Hg Septal reduction procedure within 3 months of the screening/baseline visit History of atrial fibrillation within 6 months of the screening/baseline visit or placement of ICD for secondary prevention Patients with a peak VO2 on the screening/baseline cardiopulmonary exercise test of > 80% of predicted based on age and gender Patients who require treatment with ACE inhibitors, angiotensin receptor blockers (ARBs), or renin inhibitors Known infiltrative or storage disorder such as Fabry disease, or amyloidosis Known or suspected symptomatic coronary artery diseases or evidence of prior myocardial infarction Systolic blood pressure of <100 mmHg or symptomatic hypotension during the screening/baseline period or treatment run-in period Contraindication to ARB administration or prior history of angioedema Persistent uncontrolled hypertension Other protocol-defined inclusion/exclusion criteria may apply
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Stanford
State/Province
California
ZIP/Postal Code
94305-5826
Country
United States
Facility Name
Novartis Investigative Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Novartis Investigative Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109 5271
Country
United States
Facility Name
Novartis Investigative Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Novartis Investigative Site
City
Berlin
ZIP/Postal Code
10789
Country
Germany
Facility Name
Novartis Investigative Site
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Novartis Investigative Site
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Novartis Investigative Site
City
Kiel
ZIP/Postal Code
24105
Country
Germany
Facility Name
Novartis Investigative Site
City
Athens
State/Province
GR
ZIP/Postal Code
151 23
Country
Greece
Facility Name
Novartis Investigative Site
City
Heraklion Crete
ZIP/Postal Code
711 10
Country
Greece
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Valencia
State/Province
Comunidad Valenciana
ZIP/Postal Code
46010
Country
Spain
Facility Name
Novartis Investigative Site
City
El Palmar
State/Province
Murcia
ZIP/Postal Code
30120
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
280796
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
EC1A 7BE
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Learn more about this trial

Study of Efficacy of Oral Sacubitril/Valsartan in Adult Patients With Non-obstructive Hypertrophic Cardiomyopathy

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