Clinical Trials for NAFLD & NASH in Children

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 NAFLD are being studied in children?
• What clinical studies for NAFLD are available for child participants?
• What have we learned about NAFLD in children from NIDDK-funded research?

The NIDDK conducts and supports clinical trials in many diseases and conditions, including liver 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.

Talk with your child and 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? While parents or guardians must give their permission, or consent, for their children to join a study, the children must also agree to participate, if they are capable (verbal). 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 NAFLD are being studied in children?

Researchers study many aspects of nonalcoholic fatty liver disease, or NAFLD (also referred to as metabolic dysfunction-associated steatotic liver disease, or MASLD), including

• understanding how NAFLD begins and progresses in children
• identifying genes that play a role in causing NAFLD
• improving the diagnosis of NAFLD and nonalcoholic steatohepatitis, or NASH (also referred to as metabolic dysfunction-associated steatohepatitis, or MASH), in children

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

What clinical studies for NAFLD are available for child participants?

You can view a filtered list of clinical studies on NAFLD in children that are federally funded, open, and recruiting at www.ClinicalTrials.gov. You can expand or narrow the list to include clinical studies from industry, universities, and individuals; however, the NIH 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.

What have we learned about NAFLD in children from NIDDK-funded research?

The NIDDK has supported many research projects to learn more about NAFLD and NASH in children. For example, the NIDDK’s NASH Clinical Research Network (NASH CRN) has conducted studies to advance understanding of the causes, development, complications, and treatment of NASH in children and adults.

NASH CRN studies include the Treatment of Nonalcoholic Fatty Liver Disease in Children (TONIC) trial. The TONIC trial found that the natural form of vitamin E—the type of vitamin E that comes from food sources and is not synthetic (laboratory-made)—improved the most severe form of fatty liver disease in some children. More research is needed to determine whether treatment with vitamin E causes long-term risks.

Recent NASH CRN research has explored many aspects of NAFLD and NASH in children, including

• the risk of health problems such as abnormal levels of fats in the blood and type 2 diabetes in children with NAFLD
• different subtypes of NASH that occur in children
• the relationship between NAFLD and the microbiome
• new methods to measure the severity of NAFLD without a liver biopsy
• possible treatments for NAFLD and NASH