Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A PHASE 2A, INVESTIGATOR AND SUBJECT BLINDED, SPONSOR UNBLINDED, PLACEBO-CONTROLLED, CLINICAL STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CRD-4730 IN PARTICIPANTS WITH CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA

This research study is testing a new medicine, CRD-4730, for a heart condition known as Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). CPVT can cause the heart to beat too fast or irregularly, which can be serious. This study is in an early phase, meaning it's one of the first times the medicine is being given to people. Researchers want to understand if CRD-4730 is safe to use, how well the body handles it, and if it shows any effect on the heart. Participants will either receive the new medicine or a dummy pill (placebo) to help researchers compare their results fairly. Safety is the main focus at this stage, alongside understanding how the medicine works in the body.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Cardurion Pharmaceuticals Inc.

Enrolment target

Start

14 Mar 2024

Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

What is this study about?

This study is about a heart condition called Catecholaminergic Polymorphic Ventricular Tachycardia, often shortened to CPVT. If you have CPVT, your heart can sometimes beat very fast or in an irregular pattern, especially when you're exercising or feeling stressed. This is because certain chemicals in your body (called catecholamines) can trigger these heart rhythm problems.

Researchers are testing a new medicine called CRD-4730. The main goal of this study is to find out if CRD-4730 is safe for people with CPVT and how it behaves in the body – for example, how much of it reaches the bloodstream and how long it stays there. This information helps doctors understand the right dose to use and potential side effects. This study is in an early stage (Phase 2), which means it's one of the first times this medicine is being given to a group of people to see if it could be a helpful new treatment.

Participants in the study will either receive CRD-4730 or a 'dummy' pill, called a placebo. The dummy pill looks exactly like the real medicine but doesn't contain any active drug. This is a common way to test new medicines, as it helps researchers compare the effects of the new medicine against no treatment at all. Neither you nor your study doctor will know whether you are receiving the real medicine or the dummy pill until after the study is complete. This helps to make the study results as fair and unbiased as possible.

Key takeaways

This study is testing a new medicine, CRD-4730, for CPVT patients.
It's an early-stage study focused on safety and how the medicine works in the body.
You might receive the new medicine or a dummy pill.
Your health will be closely monitored with regular check-ups and tests.
You can stop participating in the study at any time.
Participation helps further research into CPVT treatments.

Who may be eligible?

To join this study, people need to be at least 18 years old. Both men and women are welcome to take part. The study is specifically for individuals who have been diagnosed with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT).

There might be other health conditions or medicines that could prevent someone from joining, as the researchers want to make sure it's safe for everyone involved. For example, if you have other serious health problems, or are taking certain medications that might interfere with the study drug, you might not be able to participate. Pregnancy or breastfeeding would also usually prevent someone from joining a study like this.

Before you can join, the study team will carry out some health checks and ask detailed questions about your medical history and current health. This is to make sure the study is suitable and safe for you.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with CPVT?
Are you able to attend regular clinic appointments?
Are you willing to take the study medication regularly?
Are you currently not pregnant or breastfeeding?
Do you have any other serious health conditions that might prevent you from joining?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you will be given either the new medicine (CRD-4730) or a dummy pill. You'll take these pills regularly as instructed by the study team. Over the course of the study, you'll have several visits to the clinic. During these visits, doctors and nurses will check your health carefully. This will include looking for any side effects, taking blood and urine samples, checking your blood pressure and heart rate, and performing a special heart tracing called an electrocardiogram (ECG).

They will also do a physical examination to check your general health. The study team will be looking to see how your heart rhythm changes over time and how much of the study medicine is in your blood. The study will last for a specific period, and you will have follow-up appointments even after you stop taking the study medicine to ensure your continued well-being. The total duration of your involvement, including all visits and follow-ups, will be clearly explained to you.

Potential risks and benefits

Participating in a study like this might offer a potential benefit by giving you access to a new medicine that isn't widely available yet, which could help manage your CPVT. However, there are also potential risks. The new medicine might cause side effects, some of which could be serious. You might experience discomfort from the medical tests like blood draws or ECGs. It's also possible that the dummy pill won't help your condition at all. Your health will be closely monitored throughout the study. You always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

—
France
—
Italy

Common questions

What is CPVT?

CPVT (Catecholaminergic Polymorphic Ventricular Tachycardia) is a heart condition where your heart can beat very fast or irregularly, especially when you're stressed or active.

What is a 'dummy pill'?

A dummy pill, or placebo, looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the new medicine against no treatment fairly.

Will I know if I'm getting the real medicine?

No, neither you nor your study doctor will know whether you are receiving the real medicine or the dummy pill until after the study has finished. This is to ensure fair results.

Why are there so many tests?

The tests, like blood work and ECGs, are done to carefully monitor your health and safety, to see how the medicine is affecting your body, and to check for any side effects.

Can I leave the study at any time?

Yes, absolutely. You can choose to leave the study at any point, for any reason, and it will not affect the medical care you normally receive.