Clinical Trials for Hirschsprung Disease

In this section:

• Why are clinical trials with children important?
• How do I decide if a clinical trial is right for my child?
• What aspects of Hirschsprung disease is being studied in children?
• What clinical studies for Hirschsprung disease are available for child participants?

The NIDDK conducts and supports clinical trials in many diseases and conditions, including digestive diseases. The trials look to find new ways to prevent, detect, or treat disease and improve quality of life.

Why are clinical trials with children important?

Children respond to medicines and treatments differently than adults. The way to get the best treatments for children is through research designed specifically for them.

We have already made great strides in improving children’s health outcomes through clinical trials—and other types of clinical studies. Vaccines, treatments for children with cancer, and interventions for premature babies are just a few examples of how this targeted research can help. However, we still have many questions to answer and more children waiting to benefit.

The data gathered from trials and studies involving children help doctors and researchers

• find the best dose of medicines for children
• find treatments for conditions that only affect children
• treat conditions that behave differently in children than in adults
• understand the differences in children as they grow

How do I decide if a clinical trial is right for my child?

We understand you have many questions, want to weigh the pros and cons, and need to learn as much as possible. Deciding to enroll in a study can be life changing for you and for your child. Depending on the outcome of the study, your child may find relief from their condition, see no benefit, or help to improve the health of future generations.

Consider what would be expected. What could be the potential benefit or harm? Would you need to travel? Is my child well enough to participate? Parents or guardians must give their permission, or consent, for their infants to join a study. In the end, no choice is right or wrong. Your decision is about what is best for your child.

The National Institutes of Health (NIH) is committed to ensuring you get all the information you need to feel comfortable and make informed decisions. The safety of children remains the utmost priority for all NIH research studies. For more resources to help decide if clinical trials are right for your child, visit Clinical Trials and You: Parents and Children.

What aspects of Hirschsprung disease are being studied in children?

Researchers study many aspects of Hirschsprung disease in children. For example, clinical trials seek to

• better understand Hirschsprung disease and other rare disorders
• explore the role different genes play in causing Hirschsprung disease
• identify risk factors for Hirschsprung-associated enterocolitis in children with Hirschsprung disease

Watch a video of NIDDK Director Dr. Griffin P. Rodgers explaining the importance of participating in clinical trials.

What clinical studies for Hirschsprung disease are available for child participants?

You can find clinical studies on Hirschsprung disease at www.ClinicalTrials.gov. In addition to searching for federally funded studies, you can expand or narrow your search to include clinical studies from industry, universities, and individuals; however, the National Institutes of Health does not review these studies and cannot ensure they are safe. If you find a trial you think may be right for your child, talk with your child’s doctor about how to enroll.