Pediatric clinical trials to be conducted in order to investigate effectiveness and safety of study drugs in children with IBD

Living with inflammatory bowel disease (IBD) as a child is very challenging on many levels. For parents seeking treatments, it is discouraging to know that there are fewer prescription medications available to treat children with IBD.
Currently, there are twice as many IBD medications approved for adult use than for children. This means doctors don’t have as many options to treat IBD in kids.

During a clinical trial, people take an investigational medication under strict supervision by doctors and researchers. By studying the effects of existing medications on children, researchers can determine how best to use the study drug in pediatric patients. The study will help researchers better understand whether the current medications approved for adult patients can be used in children with IBD, and, if so, the accurate and safe doses for them, in order to ultimately help develop future treatment options that could improve patients’ quality of life.

However, recruitment in clinical trials is a challenging step toward the approval of potential treatment options for patients for all diseases. Nearly 80% of clinical trials fail to meet their enrollment timelines.

What families should know about pediatric clinical trials

Researchers must thoroughly study investigational drugs in the United States before doctors can prescribe them to patients of any age. The rigorous research process can take as long as 13 years to complete.

Clinical trials are among the last stages of the lengthy process. It’s important to know that participation in clinical trials is entirely voluntary, and participants can withdraw at any time. There are four phases of clinical trials that study drugs go through:

•    Phase I: During this first stage of trials, researchers look to see how the body reacts to the study drug, if the study drug is safe, and what dose of the study drug is likely to work best. This stage typically involves a small number of healthy volunteers but might occasionally involve some patients with IBD also. 
•    Phase II: If the study drug is found safe during Phase I, then it moves onto Phase II. During this stage, the study drug is given to a small number of people with IBD to see if it is effective for treating IBD and whether it causes any short-term side effects. 
•    Phase III: If there are positive results during the Phase II trial, then researchers move onto Phase II trials in which the study drug is studied in several hundred people with IBD to see how well it works compared to the standard treatment. 

If there are positive results in the first three phases of trials, then the clinical study sponsor company submits the drug to the Food & Drug Administration (FDA) for approval. 

•    Phase IV: After a study drug has been approved by the FDA, the sponsor may continue monitoring the drug to see how well it works in “real life” through Phase IV trials. 

More than drug trials

Clinical trials as part of ongoing research for IBD-related treatments are not limited to testing study medications. Various types of studies, including dietary modifications, symptom surveys, observational research and even yoga, can positively influence the types of treatments available for pediatric IBD patients.

For example, a parent in the Foundation’s Clinical Trials Community says her daughter participated in three clinical studies to alleviate her symptoms while helping other children. None of the studies involved a study drug or invasive procedure.

In one study, her daughter took children’s yoga classes and recorded her IBD symptoms on questionnaires. In a second, she completed food logs and symptom diaries for a microbiome study. The third only involved interviews where she spoke to researchers detailing her experiences with IBD symptoms.

The Crohn’s & Colitis Foundation

Drug companies, universities and disease-specific foundations, including the Crohn’s & Colitis Foundation, fund and pioneer IBD research to address the unmet needs of IBD patients. Some of our breakthrough research has resulted from what we learned during clinical trials.

Our Pediatric RISK Stratification Study has helped us predict which children will develop serious Crohn’s complications that require surgery. Additionally, our Cohort for Pediatric Translational Research in IBD (CAPTURE-IBD) effort is tracking important data on a large group of pediatric IBD patients over several years. Researchers will use this information to investigate the causes of Crohn’s disease and ulcerative colitis and help establish links between risk factors, predictive features, and health outcomes.

Find clinical trial and educational resources

The Foundation provides a comprehensive listing of IBD-related clinical trials, such as this pediatric ulcerative colitis trial, various other studies, and a robust list of resources for families and caregivers curious about clinical trials. If you’re interested in having your child join a trial, start by talking with your child’s physician. It’s vital that your child’s medical team remains in place throughout the study.

From there, the child’s caregiver will complete a digital or in-person screening with a study coordinator. This step determines if the child is eligible for participation. Next, participants may visit a clinic for lab work, follow-up and potentially other study-related testing, if needed. Every study has different requirements, and these are just a few of the participation requirements that may be part of a study.

The importance of clinical trials for pediatric IBD patients

Clinical trials offer a way to help researchers discover new, effective treatments and advance research in IBD for pediatric patients. Families interested in participating should review the resources on the Foundation’s website, consult with other families who have participated in trials, and ask open, honest questions about the process with their medical team.

Considering a clinical trial is a big decision. Take the time to learn, understand, and determine if it’s right for you and your family. 

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