Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)
This research study is testing a new medicine called vericiguat for children and young people with heart failure. Heart failure happens when the heart can't pump blood around the body as well as it should. We're looking at participants from 29 days old up to 17 years. The main aim is to see if vericiguat can improve certain markers in the blood related to heart health, compared to a placebo (a dummy medicine with no active drug). Researchers will check how well the medicine works and if it's safe. Some participants might continue into a longer-term part of the study to see the effects over time. This study hopes to find better ways to help young patients living with heart failure.
At a glance
What is this study about?
This study is about a medicine called vericiguat for children and young people who have a type of heart failure where the main pumping chamber of their heart (the left ventricle) isn't squeezing strongly enough. When the heart struggles to pump blood, it's called heart failure. We're looking for participants from just under a month old to 17 years of age.
The main goal of this study is to compare vericiguat with a 'placebo', which is a dummy medicine that looks the same but contains no active drug. We'll be checking a specific marker in the blood called NT-proBNP. This marker often goes up when the heart is under strain, so we want to see if vericiguat can help bring it down, which would suggest the heart is working better. We'll measure this at the start of the study and then again after 16 weeks.
After the initial part of the study, some participants might be able to continue taking the study medicine in a longer-term follow-up period. This helps us understand the effects of vericiguat over a longer time. By doing this research, we hope to find new and more effective ways to help young people living with heart failure.
Key takeaways
- This study is for children aged 29 days to 17 years with heart failure.
- It tests a new medicine called vericiguat against a dummy medicine (placebo).
- The aim is to see if vericiguat can improve heart health.
- Participation involves regular clinic visits for checks and blood tests.
- You can choose to withdraw your child from the study at any time.
Who may be eligible?
To join this study, children and young people must have heart failure where the main pumping chamber of their heart (the left ventricle) isn't pumping strongly enough. They need to be between 29 days old and 17 years old and weigh at least 3 kg (about 6.6 pounds). They should already be receiving stable treatment for their heart failure.
There are also some important reasons why someone might not be able to join. For example, if they are currently very unwell because of their heart failure, have certain other heart conditions, or have had a heart transplant or are waiting for one. If they've had a bad reaction to similar medicines in the past, they also wouldn't be able to take part. Females who are pregnant or breastfeeding cannot join, and those who could become pregnant would need to use effective contraception.
- Is your child between 29 days and 17 years old?
- Does your child have heart failure where the main heart pump (left ventricle) isn't working well?
- Is your child already on stable medicine for their heart failure?
- Is your child generally stable and not very unwell from their heart condition right now?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join the study, your child will either receive the study medicine (vericiguat) or a placebo (a dummy medicine). Both come as either tablets or a liquid suspension. You won't know which one your child is receiving. There will be regular visits to the clinic for check-ups, blood tests (to measure that heart marker and check for safety), and to monitor your child's overall health. These visits will happen over at least 16 weeks, and potentially longer if you choose to continue into the extended part of the study. The doctors and nurses will explain the exact schedule of visits and what each involves.
Potential risks and benefits
Locations (106)
- The Regents of the University of California - Los Angeles (UCLA Pediatrics) ( Site 0002)Los Angeles, United States· Recruiting
- Lucile Packard Children's Hospital ( Site 0040)Palo Alto, United States· Recruiting
- Loma Linda University Health System ( Site 0008)San Bernardino, United States· Recruiting
- Children's Hospital Colorado ( Site 0012)Aurora, United States· Recruiting
- Children's National Medical Center ( Site 0020)Washington D.C., United States· Recruiting
- Johns Hopkins All Children's Hospital ( Site 0029)St. Petersburg, United States· Recruiting
- Children's Healthcare of Atlanta - Arthur M. Blank Hospital ( Site 0001)Atlanta, United States· Recruiting
- Boston Children's Hospital ( Site 0035)Boston, United States· Recruiting
- C.S. Mott Children's Hospital ( Site 0033)Ann Arbor, United States· Recruiting
- Washington University-Pediatric Cardiology/ St. Louis Children's Hospital ( Site 0006)St Louis, United States· Recruiting
- Columbia University Medical Center-Pediatric Cardiology ( Site 0016)New York, United States· Recruiting
- The Children's Hospital at Montefiore ( Site 0030)The Bronx, United States· Recruiting
+94 more sites — see the official record for the full list.
Common questions
What is heart failure?
Heart failure means the heart isn't pumping blood around the body as well as it should. It doesn't mean the heart has stopped working.
What is vericiguat?
Vericiguat is a new medicine being tested to see if it can help improve heart function in people with heart failure.
What is a placebo?
A placebo is a 'dummy' medicine that looks like the real medicine but doesn't contain any active drug. It helps researchers compare the effects of the new medicine.
Will my child automatically get the new medicine?
No, your child will either receive the new medicine or a placebo. Neither you nor the study team will know which until the study is over.
How long will the study last?
The main part of the study is at least 16 weeks, but there might be an option to continue for a longer period if your child is eligible.
How to find out more
Toll Free Number
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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