Trials | Mezzion

Our clinical trials

Discovering and developing new medicines for rare diseases

FUEL (Fontan Exercise Longitudinal Assessment)

Summary:
The FUEL study explores whether Mezzion’s study drug has any benefit for helping children who have had Fontan surgery to exercise better over a 6-month treatment period. Currently there are no approved medications to help prevent aerobic capacity decline over time in this patient population.

Each individual will be randomly given either the study drug or placebo pills that they will take twice a day, every day for six months. Blood, physical measurements, measurements of vascular function (EndoPAT), echocardiograms and ECGs, and exercise function tests will be obtained at the beginning and end of the study. Some individuals in the study will also have their heart rhythm monitored before starting the study and again a few weeks after starting the study. We will also collect questionnaires about quality of life.

Eligibility:
Males or females ages 12 to 18 who have had Fontan surgery.

Primary outcome measures:
Change in exercise capacity from baseline to 26 weeks (or study completion), measured by maximal VO2 at maximum exercise effort.

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FUEL OLE (Open Label Extension)

Summary:
This includes the same individuals who participated in the FUEL study to take Mezzion’s study drug for an additional 12 months, to learn more about the possible benefit in these children.

Each individual will take the study drug twice a day, every day, for 1 year. We will also obtain blood, physical measurements, measurements of vascular function (EndoPAT), echocardiograms and ECGs, and exercise function tests at the beginning and end of the study.

Eligibility:
The same participants (males or females aged 12 to 18 who have had Fontan surgery) of the original FUEL study.

Primary outcome measures:
Change in exercise capacity from baseline to 26 weeks (or study completion), measured by maximal VO2 at maximum exercise effort.

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Evaluation of Fontan-Associated Liver Disease (FALD)

Summary:
This is an open-label, long-term, prospective study in a cohort of Fontan subjects taking the study drug daily for one year. The study will determine the scope of hepatic stiffness in the cohort by ultrasound and magnetic resonance imaging elastography and evaluate MZ101 pharmacotherapy as a potentially efficacious treatment to reduce liver stiffness.

Eligibility:
Male and female subjects 12-19 years old who are currently enrolled in the ongoing Phase 3 Open-Label Safety Study.

Primary outcome measures:
The range of liver stiffness in adolescents with single ventricle heart disease over 12 months.

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Udenafil Clinical Program

Mezzion is engaged in a clinical program that is evaluating the safety and efficacy of udenafil in adolescents with single ventricle heart disease who have undergone Fontan surgical palliation—an orphan drug indication.

The clinical program is being conducted at 28 Pediatric Heart Network (PHN) sites through the US and Canada plus additional sites in Korea. The clinical program for this orphan drug is using protocols developed by PHN, which is funded by NHLBI, an Institute of the National Institutes of Health (NIH) and approved by the FDA via its Special Protocol Assessment process.

The clinical program includes a 400-subject, randomized, double-blind, placebo-controlled trial and a long-term, open-label, safety study in approximately 300 subjects. In addition, Mezzion is evaluating the effect of udenafil on Fontan-associated liver disease—a progressive condition present in all Fontan subjects that leads to liver failure.

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