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Frequently Asked Questions

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medical team

What is a clinical research study?
How does my child’s participation make a difference?
How will being in a research study impact your family’s life?
What are the benefits and risks related to this study?
Does a child with PAH have to be in this study?
What are other options for pediatric PAH?

What is a clinical research study?

Pharmaceutical companies use clinical research studies to learn more about investigational medicines. Research studies are different than standard medical care because the purpose of these studies is to gather information about an investigational medicine. In order to conduct studies, researchers need volunteers to participate. By participating in a clinical research study, a child and his or her parent(s)/guardian(s) may help children with PAH in the future.

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How does my child’s participation make a difference?

Most medicines, drugs, medical devices, and procedures have not been tested in children. Children are often given medicines and treatments that were only tested in adults. However, children are not “little” adults. When an adult dose is adjusted to the weight of a child, it could be ineffective or even harmful. The best way to evaluate if a medicine works for children is by testing it in a pediatric clinical study.

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How will being in a research study impact your family’s life?

Parent(s)/guardian(s) have many questions when they are considering enrolling their child in a clinical research study, and your child may also want to know what he or she will go through. Families considering enrolling their child in a clinical research study should get all the information they need to feel comfortable and make informed decisions. The study doctor/study staff will be able to provide this information to you.

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What are the benefits and risks related to this study?

It is possible a child’s PAH could stop progressing while in this study, but that cannot be guaranteed. As with any research study, a child may not benefit from participation. However, a child’s participation may help children with PAH in the future.

It is possible that a participating child experiences side effects while in this study. Before starting the study, the study staff will review all known study-related risks and possible side effects with all parent(s)/guardian(s) and children.

Because research studies can affect the health of participants, all children will be monitored closely during study participation. The sponsor of this study designed a protocol that explains all study activities in detail, as well as an informed consent form, which explains the study to both the parent(s)/guardian(s) and the child.

An independent review board (IRB)/Ethic Committee (EC) has reviewed and approved this protocol. The IRB/EC requires that the protocol is followed by the study doctor and study staff exactly. The safety of children participating in the study remains the utmost priority at all times.

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Does a child with PAH have to be in this study?

No, a child with PAH does not have to be in the study. Parent(s)/guardian(s) of a child with PAH should discuss all other options with the child’s doctor.

If a child with PAH wants to be in the study, and his or her parent(s)/guardian(s) agree to study participation, the child may leave the study at any time and for any reason. However, if a child leaves the study, he or she will not be allowed to rejoin.

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What are other options for pediatric PAH?

There are multiple treatment options available to treat PAH in children. Contact your child’s doctor to discuss the best treatment plan for your child.

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