Now Enrolling:

Fontan Udenafil Exercise Longitudinal Assessment Trial - 2 (FUEL-2)

The FUEL-2 Study is a phase 3, double-blind, placebo-controlled study researching the efficacy and safety of an investigational medication, udenafil, for teenagers 12–18 years of age who had the Fontan procedure.

Who can participate?

Key inclusion criteria

Males and females with Fontan physiology

12-18 years of age, inclusive

Current antiplatelet or anticoagulant therapy

Key exclusion criteria

  • Height <132 cm.

  • Peak VO2 <45% or ≥80% of that predicted for age and

    gender.

  • Use of PDE5 inhibitors within 12 months or other pulmonary hypertension medication within 3 months.

  • Hospitalization for acute decompensated heart failure within the last 12 months.

  • Undergoing evaluation or listed for heart transplantation.

The FUEL-2 Study will include up to 436 teenagers.

Diversity among our clinical study participants is important, as racial and ethnic minorities are often not well represented in clinical studies. [1-3]

  • Although life expectancy for patients with Fontan physiology has improved, disparities in morbidity and mortality remain due to systemic inequities. [2]

  • African and Hispanic populations, in particular, tend to undergo the Fontan procedure later in life, and have limited access to long-term care. [3]

References

  1. Karamlou T, et al. J Thorac Cardiovasc Surg. 2018;155:1727-1731.

  2. Lopez KN, et al. Am Heart J. 2022;11:e025358.

  3. Soskolne G, et al. Pediatr Crit Care Med. 2012;13:218-219.

How long will I be in the FUEL-2 Study?

The study will last for up to 7 months. Before you join the study, you and your parents (or the person looking after you) will have to agree for you to take part by signing an Informed Consent Form (or ICF) or Assent Form.

You will not have any study tests done if you or your parents do not sign the ICF.

Before the study begins

  • If you decide to join the study, you will have a visit at the study center to check if the study is right for you.

  • The study doctor will want to know more about you and your condition. They will also do some tests to check your health.

  • If the study is right for you, you will receive an electronic diary (or eDiary) to record when you take the study medicine.

When you start taking the study medicine
(6 months)

  • You will have to take the study medicine as instructed, twice a day every day, for 6 months.

  • The study team will regularly check on how you are doing and if you are taking the study medicine correctly. They will call you by phone:

    • 4 times, for the first month of the study – meaning you will have a phone call once a week.

    • Then another 5 times, until you stop taking the study medicine – meaning you will have a phone call once a month.

  • At the end of this period, you will have to come back to the study center for some health checks.

After you finish taking the study medicine

  • The study team will call you, your parents, or the person looking after you by phone. This telephone call is to check if you had any changes in your health or had to take new medicines.

What will happen during the study?

You will be cared for by the study doctor and study nurses while you are taking part in this study. During your visits to the study center, the study doctor or study nurses will do some checks on your health and see how the study medicine is making you feel. These checks may include:

  • A check-up (called a “physical examination”) to check your overall health.

  • Measuring your body weight and height.

  • Urine (pee) tests.

  • Pregnancy tests (if you are a girl).

  • An exercise test.

  • Questions about your condition.

  • Questions about you, how are you feeling, other conditions you may have, and medicines you have been taking.

FAQs

  • Before new medicines can be used to treat people, doctors must make sure that the medicines work and are safe. To do this, they test the new medicines in clinical research studies, with the help of other doctors and scientists.

  • This is a study to test a new medicine for teenagers with a heart condition called single ventricle heart disease, or SVHD.

    The study doctors are looking at a possible new medicine, called udenafil. This medicine could help children and teenagers who had the Fontan procedure have more energy.

    • Udenafil works by relaxing the blood vessels and improving blood flow to the lungs. This way your blood oxygen levels are higher, and you may be less tired when exercising or during everyday activities.

    • In this study, we want to find out how well udenafil works to improve your ability to exercise and do your daily activities. We also want to know how safe udenafil is for teenagers with SVHD who had the Fontan procedure.

  • SVHD is a medical condition that some babies are born with. A heart usually has 4 chambers: 2 atria and 2 ventricles. In SVHD, the heart has only 1 ventricle. The ventricles are the main pump for the heart. They send blood out to the lungs and the rest of the body.

    • Most people with SVHD need to have multiple surgeries when they are babies to help their heart work better. One of those surgeries is called the Fontan procedure. It is done to direct the blood to the lungs instead of the heart.

    • Even after the Fontan procedure, the heart must work harder to pump enough blood and oxygen around the body. This can make it harder for children and teenagers with SVHD to do physical activities that require a lot of effort, like playing intense sports or doing challenging exercises. For this reason, you may also need to take medicines to help improve your blood flow.

  • There are different medicines to help improve your blood flow. However, some of these medicines cannot be given to teenagers or do not work the same for everyone.

    This is why it is important to research new medicine options for teenagers who had the Fontan procedure.

    We hope that what we learn in this study may help doctors treat other teenagers with SVHD who had the Fontan procedure in the future.

  • You will not be alone! More than 400 teenagers with SVHD who had the Fontan procedure, just like you, will be invited to take part in this study.

  • In this study, you will be placed by chance (like flipping a coin) in 1 of 2 groups. Depending on which group you are placed in, you will receive:

    • udenafil (the study medicine), twice a day

    • a placebo, twice a day

    The placebo is a “dummy medicine.” This means that it looks just like the study medicine but contains no active medicine. Both udenafil and the placebo can be called the “study medicine” from here on. You, your parents (or the person looking after you), and the study doctor will not know which study medicine you will receive. You will have the same chance of receiving either udenafil or the placebo.