Clinical Trials Give One Patient a Treatment He Can Stomach

Clinical Trial Drug Helps Crohn's Patient

15-year-old Sam Cox discovers one of many fish tanks in Children's Hospital Colorado.

There is a familiarity with the term Crohn's disease, but it is an illness many people don't understand.

"We had heard of Crohn's disease, but didn't know the extent of this illness until our son was diagnosed with it," said Julie Cox, whose 15-year-old son, Sam, receives treatment for the chronic illness at Children's Hospital Colorado.

Crohn's disease is an illness that causes inflammation of the digestive tract. It most commonly affects the lower part of the small intestine and can cause severe pain, diarrhea and other symptoms. According to the Crohn's and Colitis Foundation of America, an estimated 100,000 adolescents suffer from Crohn's disease nationwide.

"For more than six months Sam had flu-like symptoms and couldn't keep anything in his stomach," said Cox. "He was skin and bones, weighing 55 pounds at four-feet, seven inches."

Sam gets a gut check

Cox said it was frightening to not know what was wrong with her son. Sam's primary care doctor ran blood work and referred him to Edward Hoffenberg, MD, professor of pediatrics, and director of the Inflammatory Bowel Diseases Center of the Digestive Health Institute at Children's. A colonoscopy and endoscopy confirmed a diagnosis of Crohn's disease.

Dr. Hoffenberg diagnosed Sam in April 2007 and immediately started a personalized treatment plan. Within a few months Sam was back to a healthy weight. The maintenance medications he tried over the next two years seemed to stabilize his condition, but in September 2009 he was sick again - down to 80 pounds - weak and tired all the time.

"As a parent it's so difficult to watch your child go through something like this," said Cox. "We wanted to try anything to help him feel better."

That's when Dr. Hoffenberg suggested Sam enroll in a clinical trial, an investigational treatment option.

Clinical trial investigates Crohn's drug for children

"Many clinical trials have proven to be wonderful choices for participants, better than anything currently available," said Dr. Hoffenberg. "I have carefully chosen clinical trials in which to participate, selecting only those where the trial fills a need for medical therapies."

Children's fastidiously selects which studies are conducted. An ethics review committee approves each trial and a team of dedicated investigators are very involved with each study population. The investigators monitor the treatment and ensure the trial is administered in a safe environment where patients and their families feel comfortable. Participation is always voluntary, and caregivers and patients are thoroughly informed about the trial and requirements.

"Through laboratory and clinical research, Children's Digestive Health Institute in partnership with the University of Colorado School of Medicine continues to develop and test cutting-edge treatments and cures that will improve the lives of children with digestive and liver problems," said Ronald Sokol, MD, vice chair of pediatrics, chief, section of pediatric gastroenterology, hepatology and nutrition and the Digestive Health Institute.

The Food and Drug Administration (FDA) has approved the drug from Sam's clinical trial for use in adults. The study objective is to determine the safety and efficacy of this drug in children six to 17 years of age who suffer from Crohn's disease.

"Many of the medications approved by the FDA are approved for adults; however, these medications are not necessarily appropriate for children," said Sue Brantz, RN, BSN, CCRC, research coordinator. "Children are not mini adults; they metabolize differently, and it's very important that we treat children as children, ensuring that all medications are safe and efficacious for them."

Sam is the first patient at Children's to enroll in this trial so far, although Children's has screened other patients. Thirty to 40 sites are conducting this particular study with hope to enroll about 200 patients. Each site, including Children's, intends to enroll up to five children. Once patients are screened, enrolled, and show a response, a one-year maintenance period follows, with an additional 12-month follow-up period.

Sam started the clinical trial study at the end of January 2010, and according to his mom, his condition has greatly improved.

"From our perspective as parents, the trial study has been fantastic, especially the staff and Dr. Hoffenberg," said Cox. "Sam has energy, he's social, focused on school and his personality is back - he's a teenager again."

Learn more about digestive, pancreas and nutrition conditions we treat.

Scientists and clinicians research disease and manage care

Ongoing laboratory research and clinical trials related to pediatric gastrointestinal and liver diseases contribute to pediatric medical advancements and education for the next generation of pediatricians and pediatric specialists.

An example of this research is a multi-center study funded by the National Institutes of Health (NIH) and the Cystic Fibrosis (CF) Foundation to identify a means to identify children with CF who may develop cirrhosis, or severe scarring of the liver. Cirrhosis occurs in about seven percent of children with CF and develops by 15 years of age. The study is led by Michael Narkewicz, MD, medical director of Children's Liver Center and the Hewit/Andrews Chair in Pediatrics Liver Diseases.

"This study is looking at whether the use of abdominal ultrasound and blood biomarkers can identify children with CF who will develop cirrhosis. This research is necessary before we can begin studying drugs that are available today that might prevent cirrhosis," said Dr. Narkewicz. "It is an example of collaborative research supported by Children's Digestive Health Institute's Liver Center, CF Center, radiology department, NIH funding and the CF Foundation."