ÃÛÌÒAPP

• 14-year-old Robert Davidson from Gateshead is the first patient in the UK to be recruited to the VALOR trial
• ÃÛÌÒAPP Hospitals is one of only two hospitals in the UK running the study.

The Freeman Hospital, part of ÃÛÌÒAPP Hospitals, has recruited the first patient in the UK to a children’s heart study.

14-year-old Robert Davidson who lives with a rare muscle-wasting disease, Duchenne muscular dystrophy (DMD), is the first patient to be enrolled into the VALOR trial.

The VALOR study is looking to see if an investigational medicine (Vericiguat) can be safely given to infants, children, and teenagers to improve heart failure.

Heart failure is a serious disease that means the heart does not pump enough blood to the body.

In children, heart failure can be due to a primary heart muscle disease (cardiomyopathy), secondary to congenital (from birth) heart disease, or it can be associated with other medical conditions.

While heart failure can be managed, it increases the chances of hospitalisation and sadly, in some cases, it can lead to death.

Vericiguat works by relaxing the blood vessels in the lungs to allow blood to flow easily. Previous studies in adults have found that it lowers the chance of hospital admission and death from heart failure, however the medication has not been tested specifically in children.

In the study, Vericiguat will be trialled in children aged 28 days to 18 years of age. The study is double-blinded and randomised, meaning patients will be randomly allocated to receive Vericiguat or the placebo dose, neither of which will be known to the patient or clinical team.

Dr Abbas Khushnood, Principal Investigator for the study and Consultant Paediatric Cardiologist and Transplant physician at the Freeman Hospital, said:

“Heart failure is complex, and it can be incredibly difficult for children and their families to live with.

“There is currently very little evidence to show which medications work specifically for children as most clinical trials to date have focussed on adults.

“The VALOR trial marks an important step in building a body of evidence to help clinical teams decide the most effective treatment for children with heart failure.”

Patient story
Many medical conditions in children can be associated with cardiomyopathy, as is the case for patients with DMD, like Robert.

The condition is caused by a fault on the dystrophin gene, a protein that protects muscles and helps them to function and repair.

DMD almost always affects boys and is carried down on the mother’s side. It eventually affects all muscles in the body, including the heart muscle which becomes weak and unable to pump blood around the body effectively.

Mum Lindsay first took oldest son Angus to the doctor when she and husband, Stuart, noticed he was having problems running, jumping, and climbing the stairs. After having an urgent blood test, she was told by her GP that Angus has DMD.

Robert also needed to be tested, and the family was told the devastating news that he, too, had the condition.

Lindsay said:

“We received a double blow when we found out both Robert and Angus had DMD. Knowing the boys have a condition that reduces life expectancy is devastating, but our family and friends have been an incredible support to us.

“We are very supportive of clinical trials and the difference they can make to people living with conditions such as Duchenne muscular dystrophy.

“Anything that could contribute to keeping the heart as healthy as possible and give people with DMD the chance of living a healthier, longer life is worth doing in my eyes.”

The trust’s paediatric clinical research team is running VALOR and supporting Robert throughout the trial. 16-year-old Angus is awaiting screening for consideration of enrolment to the trial.

Robert and Angus are under the care of Dr Khushnood and are regularly monitored for signs of heart failure.

Angus is enjoying his time at college and Robert attends school where he particularly likes science and geography. He also likes taking his dog for walks, playing videos games, and powerchair football.

Dr Khushnood added: “Clinical trials such as this are vital to ensuring children living with heart failure can have a brighter, healthier future.”