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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04426578




Registration number
NCT04426578
Ethics application status
Date submitted
14/05/2020
Date registered
11/06/2020

Titles & IDs
Public title
Role of Perhexiline in Hypertrophic Cardiomyopathy
Scientific title
Randomised Controlled Trial of pErhexiline on regreSsion Of Left Ventricular hypErtrophy (LVH) in Patients With Symptomatic Hypertrophic CardioMyopathy (RESOLVE-HCM)
Secondary ID [1] 0 0
HCM2020-01
Universal Trial Number (UTN)
Trial acronym
RESOLVE-HCM
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypertrophic Cardiomyopathy 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Perhexiline
Other interventions - Placebo

Experimental: Perhexiline -

Placebo comparator: Placebo -


Treatment: Drugs: Perhexiline
All eligible and consented patients will be randomised to initiation of perhexiline 100mg once daily or identical placebo. After 4 days of treatment, a blood sample will be collected to determine plasma perhexiline concentrations: timing of the sample need not be "trough" in view of the long-acting nature of perhexiline. Depending on the blood results, patients might require as little as 50mg/week (slow metabolisers) or as much as 600mg/day (ultra-rapid metabolisers). The initial sample will be utilized primarily to detect presence of hydroxylated metabolite: patients in whom perhexiline is detected in the absence of metabolite will be designated "slow metabolisers" and will have their dosage reduced to 50 mg/week in the first instance. Repeat assay at 30 days will be utilized for individual finer dose titration based on dose adjustment table. Paired dosage adjustment in placebo-treated patients will be performed to avoid unblinding.

Compliance will be assessed by capsule count.

Other interventions: Placebo
All eligible and consented patients will be randomised to initiation of perhexiline 100mg once daily or identical placebo. After 4 days of treatment, a blood sample will be collected to determine plasma perhexiline concentrations: timing of the sample need not be "trough" in view of the long-acting nature of perhexiline. Depending on the blood results, patients might require as little as 50mg/week (slow metabolisers) or as much as 600mg/day (ultra-rapid metabolisers). The initial sample will be utilized primarily to detect presence of hydroxylated metabolite: patients in whom perhexiline is detected in the absence of metabolite will be designated "slow metabolisers" and will have their dosage reduced to 50 mg/week in the first instance. Repeat assay at 30 days will be utilized for individual finer dose titration based on dose adjustment table. Paired dosage adjustment in placebo-treated patients will be performed to avoid unblinding.

Compliance will be assessed by capsule count.
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Left Ventricular Hypertrophy (LVH)
Timepoint [1] 0 0
12 months post baseline
Secondary outcome [1] 0 0
Change in Left Ventricular (LV) mass
Timepoint [1] 0 0
12 months post baseline
Secondary outcome [2] 0 0
Change in oxygen-sensitive Cardiac Magnetic Resonance
Timepoint [2] 0 0
12 months post baseline
Secondary outcome [3] 0 0
Change in left ventricular diastolic function
Timepoint [3] 0 0
12 months post baseline
Secondary outcome [4] 0 0
New York Heart Association (NYHA) functional classification
Timepoint [4] 0 0
12 months post baseline
Secondary outcome [5] 0 0
Canadian Cardiovascular Society (CCS) functional class
Timepoint [5] 0 0
12 months post baseline
Secondary outcome [6] 0 0
Quality of life assessment
Timepoint [6] 0 0
12 months post baseline
Secondary outcome [7] 0 0
Major adverse event on heart failure related hospitalisations
Timepoint [7] 0 0
Monitored over the 12 months period
Secondary outcome [8] 0 0
Major adverse event on arrhythmic events
Timepoint [8] 0 0
Monitored over the 12 months period
Secondary outcome [9] 0 0
Major adverse event on abnormal liver function test
Timepoint [9] 0 0
Liver function tests at baseline, 1 month, 6 months and 12 months
Secondary outcome [10] 0 0
Major adverse event on sudden cardiac death
Timepoint [10] 0 0
Monitored over the 12 months period

Eligibility
Key inclusion criteria
1. Left Ventricular Ejection Fraction (LVEF) =/> 55% by echocardiography or CMR during the screening period or within 6 months prior to study entry
2. Current / prior symptom(s) of HCM (New York Heart Association [NYHA] functional class II or class III, Canadian Cardiovascular Society [CCS] grade II or grade III) and requiring treatment with ß-blockers and /or non-dihydropyridine calcium antagonists and / or disopyramide for at least 30 days prior to study entry
3. Structural heart disease as evidenced by interventricular septal thickness of (= 15 mm) on echocardiography or CMR in the absence of abnormal loading conditions
4. Elevated N terminal pro-brain natriuretic peptide (NT-proBNP), >125 pg/ml
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any prior echocardiographic or CMR measurement of LVEF <55%
2. Current acute decompensated heart failure requiring hospitalisation and / or augmented medical therapy
3. Cardiac surgery or catheter-based septal reduction therapy planned or having occurred within the past 1 year
4. Patients with a non-CMR conditional pacemaker / implantable cardioverter-defibrillator device
5. History of a known chronic liver disease, peripheral neuropathy, recurrent hypoglycemia
6. Serum bilirubin, alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, or lactate dehydrogenase > 2.0 times upper limit of normal
7. Previous adverse reaction to perhexiline at therapeutic plasma levels of the drug
8. Concomitant use of amiodarone, ranolazine or trimetazidine
9. Life-threatening or uncontrolled dysrhythmia
10. Contraindications to CMR, gadolinium, adenosine

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2022
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Flinders Medical Centre - Adelaide
Recruitment postcode(s) [1] 0 0
5042 - Adelaide

Funding & Sponsors
Primary sponsor type
Other
Name
Flinders University
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Joseph Selvanayagam
Address 0 0
Flinders Medical Centre
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Joseph Selvanayagam
Address 0 0
Country 0 0
Phone 0 0
+61882045619
Fax 0 0
Email 0 0
joseph.selva@sa.gov.au
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04426578