Study to Evaluate Sotatercept (MK-7962) in Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-008)
This study is investigating a new medication, sotatercept, for children and teenagers aged 1 to 17 who have a specific type of pulmonary arterial hypertension (PAH). PAH is a serious condition where blood vessels in the lungs become narrow, making it hard for the heart to pump blood. The main goals are to check if sotatercept is safe for young people and how well their bodies handle the medicine over 24 weeks. Participants will continue to receive their standard PAH treatments while taking part. This is an early-stage study, meaning we are still learning about this medicine, and it's not designed to prove if the medicine works better than current treatments, but rather to understand its safety and how it behaves in children.
At a glance
What is this study about?
This study is looking into a new medicine called sotatercept for children and teenagers between 1 and 17 years old who have a serious lung condition called pulmonary arterial hypertension. PAH happens when the blood vessels in the lungs become narrow and stiff, making it difficult for the heart to pump blood through them. This can cause the heart to work too hard.
The main reasons for this study are to see if sotatercept is safe for young people and to understand how their bodies process the medicine over 24 weeks. Participants will continue taking their usual PAH medicines during the study. This is an important step in developing new treatments for PAH in children. By understanding how sotatercept behaves in younger patients, doctors can learn more about its potential use.
It's important to know that this is a 'Phase 2' study. This means it's an early step in testing a new medicine. We're not yet trying to prove if the medicine works better than existing treatments, but rather focusing on its safety and gathering initial information on how it works in the body. This kind of research is vital for bringing new and potentially helpful medicines to those who need them.
Key takeaways
- This study is for children aged 1-17 with a specific type of PAH.
- It's testing a new medicine called sotatercept for safety and how the body handles it.
- Participants will continue their usual PAH treatments.
- This is an early-stage study (Phase 2) to gather important information.
- Participation involves regular clinic visits for monitoring.
- You can withdraw from the study at any time without affecting your child's care.
Who may be eligible?
To join this study, a child must have a confirmed diagnosis of a specific type of pulmonary arterial hypertension (PAH), which would have been diagnosed through a special heart test called a right heart catheterisation. This diagnosis must have happened before screening for the study. The types of PAH covered include those that run in families, those caused by certain drugs, or PAH linked to other conditions like connective tissue disease.
Children need to be between 1 and 17 years old. They must also already be on a steady dose of their usual PAH medicines, such as those that relax blood vessels or help open them up. These medicines must have been taken consistently without changes for a while before the study starts.
There are also important rules about avoiding pregnancy during the study and for some time after the last dose of the study medicine, for both male and female participants. This is because we don't know the effects of the study medicine on an unborn baby. For example, specific contraception methods or agreement to not have PIV during this period would be necessary. Also, participants cannot donate blood or sperm during the study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child aged between 1 and 17 years old?
- Does your child have a confirmed diagnosis of a specific type of PAH?
- Is your child currently taking a steady dose of their usual PAH medicines?
- Are you able to follow specific rules about avoiding pregnancy during and after the study for your child?
- Has your child had their PAH diagnosis confirmed by a special heart test called a right heart catheterization?
What does participation involve?
If you or your child decides to take part, they will receive the study medicine, sotatercept, for 24 weeks, while continuing all their usual PAH treatments. During this time, there will be regular visits to the clinic so the study team can monitor their health, perform necessary tests, and make sure the medicine is being taken safely. These tests might include blood samples, physical examinations, and other checks related to their heart and lungs. After the 24 weeks of treatment, there will be some follow-up visits to ensure your child is doing well and to collect final information. The exact number of visits and tests will be explained in detail by the study doctor, and the total duration of your child's involvement would be beyond the 24 weeks of treatment, including follow-up time.
Potential risks and benefits
Locations (35)
- The Regents of the University of California - Los Angeles (UCLA Pediatrics) ( Site 1606)Verified postcodeLos Angeles, United States· Recruiting
- Stanford University School of Medicine ( Site 1603)Verified postcodePalo Alto, United States· Recruiting
- UCSF Benioff Children's Hospital San Francisco ( Site 1611)Verified postcodeSan Francisco, United States· Recruiting
- Children's Hospital Colorado ( Site 1609)Verified postcodeAurora, United States· Recruiting
- Children's National Medical Center ( Site 1600)Verified postcodeWashington D.C., United States· Recruiting
- Cincinnati Children's Hospital Medical Center ( Site 1602)Verified postcodeCincinnati, United States· Recruiting
- Children's Hospital of Philadelphia (CHOP) ( Site 1608)Verified postcodePhiladelphia, United States· Recruiting
- Monroe Carell Jr. Children's Hospital ( Site 1601)Verified postcodeNashville, United States· Completed
- Seattle Children's Hospital ( Site 1605)Verified postcodeSeattle, United States· Recruiting
- Children's Wisconsin ( Site 1610)Verified postcodeMilwaukee, United States· Recruiting
- The Children's Hospital at Westmead ( Site 0001)Verified postcodeWestmead, Australia· Recruiting
- Clinica Somer-Unidad de Investigacion y Docencia ( Site 0205)Verified postcodeRionegro, Colombia· Recruiting
Common questions
What is Pulmonary Arterial Hypertension (PAH)?
PAH is a serious lung condition where the blood vessels in the lungs become narrow, making it hard for the heart to pump blood through them.
What is the purpose of this study?
This study aims to find out if the new medicine, sotatercept, is safe for children with PAH and how their bodies handle it over 24 weeks, alongside their usual treatments.
Will my child stop their current PAH medicines if they join?
No, your child will continue to take their usual PAH medicines during the study. Sotatercept would be an additional treatment.
What does 'Phase 2' study mean?
Phase 2 means it's an early stage of testing a new medicine. We're focusing on safety and initial understanding, not yet proving it works better than other treatments.
Can we stop participating at any time?
Yes, you can choose to withdraw your child from the study at any point, and it will not affect their medical care.
How to find out more
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Always speak to your GP or specialist before deciding to take part in a study.
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