Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure

Cardiomyopathy, Hypertrophic Clinical Trial

Official title:

A Phase 2, Multi-Center, Open-Label, Ascending Dose Study on the Efficacy, Safety and Tolerability of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure With Preserved Left Ventricular Function

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02862600
• Other study ID #: HML-PHX-005
• Status: Recruiting
• Phase: Phase 2
• First received: August 8, 2016
• Last updated: February 16, 2017
• Start date: August 2016
• Est. completion date: August 2017

Study information

• Verified date: February 2017
• Source: Heart Metabolics Limited
• Contact: Heart Metabolics Call Center
• Phone: 1-800-206-2434
• Email: info[@]heartmetabolics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 33
• Est. completion date: August 2017
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Key Inclusion Criteria:

• Hypertrophic cardiomyopathy with symptoms of moderate-to-severe heart failure

• Left ventricular hypertrophy with maximum LV wall thickness = 15 mm

• Left ventricular ejection fraction = 50%

• Able to perform exercise testing but unable to exceed 75% of the predicted age-adjusted maximum level

Key Exclusion Criteria:

• CYP2D6 Poor Metabolizer (PM) status

• History of a known chronic liver disease

• ALT, AST, alkaline phosphatase, or LDH > 1.5 x upper limit of normal

• Total Bilirubin > 2.0 x upper limit of normal

• Severe LV outflow obstruction

• Asymptomatic patients or cardiomyopathy-related criteria as per protocol

• QT interval related criteria as per protocol

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Hypertrophic
• Cardiomyopathy, Hypertrophic, Familial
• Heart Failure
• Hypertrophy

Intervention

Drug:
• Perhexiline
Period 1 (Weeks 1-8) and Period 2 (Weeks 9-16): dose titrated to two different plasma levels of perhexiline

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Heart Metabolics Limited

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline of VO2MAX		end of Period 2 (Week 16)
Secondary	Change from baseline of VO2MAX		end of Period 1 (Week 8)
Secondary	Change from baseline of VO2MAX		end of Period 1 (Week 8) compared to end of Period 2 (Week 16)
Secondary	Change from baseline in the six-minute walk test		end of Period 1 (Week 8)
Secondary	Change from baseline in the six-minute walk test		end of Period 2 (Week 16)
Secondary	Change from baseline in the six-minute walk test		end of Period 1 (Week 8) compared to end of Period 2 (Week 16)
Secondary	Safety measures to include assessments of adverse events, vital signs, ECGs, and laboratory assay results		during Period 1 (Weeks 1-8) compared to during Period 2 (Weeks 9-16)