Not yet recruitingPhase IIInterventional

A trial to evaluate the safety and efficacy of treatment with azacitidine in patients with symptomatic non-obstructive hypertrophic cardiomyopathy

This research is investigating a new treatment called azacitidine for a heart condition called non-obstructive hypertrophic cardiomyopathy (nHCM). HCM is a common inherited heart problem where the heart muscle thickens; nHCM means the blood flow isn't blocked. While treatments exist for other types of HCM, options for nHCM mostly manage symptoms rather than the underlying disease. Azacitidine is already approved for some blood cancers and has shown promise in improving heart health markers. This study aims to find the right dose and then check how safe and effective azacitidine is for people with symptomatic nHCM, hoping to offer a new way to treat this condition.

At a glance

Status

Not yet recruiting

Phase

Phase II

Sponsor

Belfast Health and Social Care Trust

Enrolment target

Start

30 Jun 2026

Estimated completion

28 Apr 2028

Symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM)

What is this study about?

Heart conditions can be worrying, and one common inherited type is called hypertrophic cardiomyopathy (HCM). In HCM, the heart muscle becomes unusually thick. There are two main types: obstructive (oHCM) where the thickening blocks blood flow, and non-obstructive (nHCM) where it doesn't. While there have been good advances in treating oHCM, current treatments for nHCM mainly help ease symptoms rather than targeting the disease itself. This can be frustrating for people living with the condition.

This study, called AZTEC, is looking into a drug called azacitidine as a potential new treatment for nHCM. Azacitidine is already used to treat certain blood cancers, and early research suggests it might also improve how the heart works and protect it from further damage. The aim is to see if this existing drug could offer new hope for people with nHCM.

This research is important because it could lead to new ways to treat nHCM, aiming to do more than just manage symptoms. By carefully testing azacitidine, the study hopes to find a safe and effective dose, and ultimately improve the health and quality of life for those with this heart condition.

Key takeaways

Investigating azacitidine for non-obstructive hypertrophic cardiomyopathy (nHCM).
Current nHCM treatments mainly manage symptoms, not the disease itself.
Azacitidine is already used for blood cancers and may help heart health.
Study has two phases: finding the right dose, then comparing to standard care.
Participation involves injections, various heart checks, and blood tests.
Potential risks include common drug side effects; benefits include new treatment development.

Who may be eligible?

This study is looking for adults aged 18 to 75 who have been diagnosed with hypertrophic cardiomyopathy (HCM) and have symptoms. Specifically, your heart muscle should show a certain degree of thickening.

You also need to be experiencing noticeable symptoms of your HCM in your daily life, as defined by specific medical classifications (NYHA functional class II/III). There are also some specific blood test results and heart measurements that will need to be within a certain range to be part of the study.

There are also reasons you might not be able to join, such as having had certain cancers recently, being pregnant or breastfeeding, or having specific problems with your blood counts or kidney/liver function. You also cannot be taking certain other experimental medications or if you or your partner are of childbearing potential and unwilling to use specific birth control methods.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 75 years old?
Do you have a diagnosis of hypertrophic cardiomyopathy (HCM) with a thickened heart muscle (15mm or 13mm with family history)?
Are you experiencing symptoms of your HCM (NYHA functional class II/III)?
Have you had certain blood tests (like NT-proBNP) and heart measurements (like LVEF and LVOT gradient) that meet specific study criteria?
Do you have no history of certain cancers in the last 5 years (excluding common skin cancers)?
Are you able to use acceptable birth control methods if you or your partner could become pregnant?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, it will run until December 2028. It has two main parts. The first part (Phase IIa) will involve a small group of up to 24 participants. You would receive azacitidine injections twice a week for 16 weeks. The dose might change depending on how well earlier participants tolerate it. You'll have various check-ups before and after this treatment, including a 6-minute walk test, heart scans (echocardiogram and cardiac MRI), and regular blood tests. You'll also fill out questionnaires about your symptoms.

Once the best dose is found, the second part (Phase IIb) will involve up to 48 participants. You'll be randomly assigned to one of two groups: either you'll receive the azacitidine injections twice a week for 16 weeks (just like in Phase IIa, with all the same tests), or you'll continue with your usual medical care. If you're in the usual care group, you'll still have physical exams, blood tests, and heart health checks at specific times, but won't receive the study drug. Throughout the study, your health will be closely monitored, and any side effects will be recorded.

Potential risks and benefits

Taking part in new research is very important for finding better treatments, especially for conditions like hypertrophic cardiomyopathy where options can be limited. However, because azacitidine is a new treatment for this particular heart condition, we can't be completely sure of the outcome, and there's no guarantee it will help you. Potential side effects could include an increased risk of infections, bleeding, allergic reactions, changes in appetite or sleep, dizziness, headaches, fluid around the heart, changes in blood pressure or breathing, stomach problems (like constipation, diarrhoea, or sickness), issues with kidney or liver function, muscle aches, skin irritation, and general symptoms like fever, tiredness, and discomfort where injections are given. All these potential side effects will be carefully watched and recorded. Remember, you can choose to leave the study at any time.

Locations (1)

Belfast City Hospital
Approximate
Belfast, Northern Ireland

Common questions

What is non-obstructive hypertrophic cardiomyopathy (nHCM)?

It's an inherited heart condition where the heart muscle is thickened, but this thickening doesn't block the blood flow out of the heart.

What is azacitidine?

It's a medication already approved for treating certain blood cancers. This study explores its potential use for nHCM.

How will I receive the treatment?

If you are in the treatment group, you will receive azacitidine as injections twice a week for 16 weeks.

Where is this study taking place?

The study is being run from the Northern Ireland Inherited Cardiac Conditions Services, at Belfast City Hospital in the UK.

Who is paying for this research?

The study is funded by the British Heart Foundation and the Heart Trust Fund in the UK.

How to find out more

Megan Campbell

AZTEC@nictu.hscni.net +44 (0)28961 51447 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.