Condition category
Circulatory System
Date applied
08/01/2015
Date assigned
09/01/2015
Last edited
27/01/2015
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Background and study aims
Hypertrophic cardiomyopathy (HCM) is the most common inherited heart condition. One in every 500 people have the condition and there are one million sufferers in Europe. It is a disorder of the heart muscle (myocardium) itself results in
heart muscle thickening (hypertrophy) and scarring (fibrosis). This condition can cause disturbances in heart rhythms which can cause sudden death, indeed HCM is the leading cause of sudden death in young (<35 years old) people. HCM hearts process energy inefficiently, and progressive heart failure can also develop. There are currently no treatments that alter the natural history of the disease. It is not clear how the faulty gene causes the muscle to become thickened, however current research suggests that it is a problem with our body cells not being able to use energy properly. Trientine is a medication used to treat Wilson disease, a rare disorder which leads to excess copper accumulation and tissue damage since 1969. It also acts on energy usage to improve it. Trientine has been found to reduce heart muscle hypertrophy in patients with diabetes. The aim of this study is to investigate whether Trientine will reduce the thickening of the heart in HCM patients, improve its energy efficiency and therefore represent the first disease modifying therapy in HCM.

Who can participate?
Adults (aged at least 18) with HCM.

What does the study involve?
Partipicants are randomly allocated into one of two groups. Those in group 1 receive their usual care. Those in group 2 are given trientine for 6 months. All patients undergo cardiac magnetic
resonance (CMR) imaging at the start of the study and then after 6 months of therapy. CMR assesses how the heart uses its energy, myocardial hypertrophy and fibrosis.

What are the possible benefits and risks of participating?
Not provided at time of registration

Where is the study run from?
Wythenshawe Hospital (UK)

When is the study starting and how long is it expected to run for?
October 2014 to October 2015

Who is funding the study?
Medical Research Council (UK)

Who is the main contact?
Dr Anna Reid

Trial website

Contact information

Type

Scientific

Primary contact

Dr Anna Reid

ORCID ID

Contact details

Wythenshawe Hospital
Southmoor Road
Wythenshawe
Manchester
M23 9LT
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

17776

Study information

Scientific title

Copper Chelation in Hypertrophic Cardiomyopathy: Open-label pilot study of Trientine in patients with hypertrophic cardiomyopathy

Acronym

Study hypothesis

We hypothesise that trientine will lead to left ventricular mass regression in hypertrophic cardiomyopathy. Furthermore, we hypothesis that improvement in myocardial energetics is associated with, or indeed causative of, this LV regression.

Ethics approval

NRES Committee North West- Liverpool East, 26/06/2014, ref: 14/NW/1015

Study design

Non-randomised; Interventional

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

Topic: Cardiovascular disease; Subtopic: Cardiovascular (all Subtopics); Disease: Cardiovascular

Intervention

Using trientine to assess if it improves fitness and heart function to improve treatment options for HCM.

Intervention type

Other

Phase

Phase IV

Drug names

Primary outcome measures

1. Evaluate myocardial energetics
2. To evaluate myocardial energetics, as measured using cardiac MRI, in HCM patients undergoing treatment

Participants will receive Trientine for 6 months. Outcomes will be measured prior to starting treatment, and at the end of treatment.

Secondary outcome measures

N/A

Overall trial start date

01/10/2014

Overall trial end date

01/10/2015

Reason abandoned

Eligibility

Participant inclusion criteria

1. Male or female > 18 years of age
2. Females will be non-pregnant and non-lactating with no intention of pregnancy during study treatment (see point 6)
3. Confirmed diagnosis of HCM in line with 2011 ACCF / AHA consensus document
4. Positive genotype
5. LV ejection fraction = 50%
6. Women of childbearing potential (not >1 year post-menopausal) must agree to use one of the following acceptable birth control methods:
6.1. True complete abstinence when this is in line with the preferred and usual lifestyle of the subject
6.2. Surgical sterilization of either the female subject in study (e.g., bilateral tubal ligation) or of her male partner (vasectomy with documented azoospermia) if he is the sole partner of that subject
6.3. Established progesterone-only hormonal contraception (implantable, patch, oral or intramuscular [IM]) administered for at least one month prior to study medication administration
6.4. Double barrier method: condom and occlusive cap (diaphragm) with spermicidal foam/gel/film/ cream/suppository

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Planned Sample Size: 20; UK Sample Size: 20

Participant exclusion criteria

1. History of any other cardiovascular disorder, including aortic stenosis, aortic coarctation, hypertension, renal artery stenosis, atrial fibrillation
2. NYHA Class III / IV heart failure
3. Diabetes Mellitus
4. Contraindication to magnetic resonance imaging (MRI) scanning (including claustrophobia)
5. Known hypersensitivity to Trientine or excipients
6. Known hypersensitivity to Gadolinium-based contrast agent
7. eGFR < 50ml/min/1.73m2
8. BMI > 40kg/m2
9. History of significant malabsorption
10. Copper deficiency at baseline
11. Iron deficiency at baseline
12. Haemoglobin < 10g/dL
13. Unresolved haematological disorder
14. Severe hepatic impairment
15. Untreated thyroid disease
16. Autoimmune disorders/connective tissue disease
17. Drug or alcohol abuse
18. Pregnancy/breast-feeding. Women of childbearing potential (not >2 years post- menopausal and/or not surgically sterilised) must have a negative blood serum pregnancy test, performed at visit 1 prior to administration of study medication
19. Any clinically significant or unstable medical or psychiatric condition that would interfere with the patient’s ability to participate in the study
20. Any other condition, which in the opinion of the research team, may put participants at risk during the study, or which may affect the outcome of the study
21. New medication within the preceding month of the study (excluding short term prescriptions)
22. Participation in another study involving an investigational product in the previous 12 weeks

Recruitment start date

06/01/2015

Recruitment end date

01/10/2015

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Wythenshawe Hospital
Southmoor Road Wythenshawe
Manchester
M23 9LT
United Kingdom

Sponsor information

Organisation

University Hospital of South Manchester NHS Foundation Trust

Sponsor details

Southmoor Road
Wythenshawe
Manchester
M23 9LT
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

Medical Research Council

Alternative name(s)

MRC

Funding Body Type

private sector organisation

Funding Body Subtype

other non-profit

Location

United Kingdom

Results and Publications

Publication and dissemination plan

To be confirmed at a later date

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes