The first approved pediatric clinical trial of Tremfya (guselkumab), a medication approved to treat plaque psoriasis, psoriatic arthritis, Crohn’s disease and ulcerative colitis in adults, is yielding promising results under the supervision of researchers from the Indiana University School of Medicine.
Ulcerative colitis is a chronic inflammatory bowel disease in which the lining of the large intestine and rectum becomes inflamed. Symptoms often include abdominal pain, bloody diarrhea, fatigue and weight loss. Patients may often need blood transfusions, and in cases where treatments prove ineffective, they risk the loss of their colon.
Two pediatric participants have qualified for the clinical trial, one in the United States and the other in Europe. The enrollment site at the IU School of Medicine and Riley Children’s Health has become the first location in the world to welcome a pediatric patient into the Tremfya pediatric trial.
Due to vast experience with multicenter clinical trials, industry-sponsored drug studies and a reputation for well-established research conducted by gastroenterologists and research coordinators, the IU-Riley site is a tertiary referral center for children in Indiana and neighboring states needing advanced medical care, and the Pediatric Gastroenterology group is a well-positioned leader for the trial.
Marian Pfefferkorn, MD, professor of clinical pediatrics, director of endoscopy for the Division of Pediatric Gastroenterology, Hepatology and Nutrition within the IU School of Medicine and a pediatric gastroenterologist at Riley Children’s Health, is the principal investigator of the pediatric Tremfya trial.
Initially, the patient who is now enrolled in the Tremfya trial was treated with IV-steroid and infliximab medicine treatment. Although the administration of these steroids has proven effective in many cases, the treatment was not effective for this patient and led to the continuation of colitis symptoms. Normally, the next step after ineffective medical treatment is for a patient to undergo a colectomy, an operation where the colon is removed. Fortunately, an alternative was presented in the form of the pediatric trial for Tremfya.
“With team effort, especially the research coordinator identifying that this patient would be a good candidate for this clinical trial, the pieces just fell into place, and we all agreed that it was worth a try to save the patient's colon,” Pfefferkorn said.
As principal investigator of the trial, Pfefferkorn is involved from the moment the patient is chosen through the protocol of observing if the treatment is benefiting the patient and determining if future use of the medication would be applicable to other young patients.
“And so far, yes, she is responding and we've avoided surgery for her,” Pfefferkorn said.
With three loading doses administered intravenously or subcutaneously, followed by maintenance therapy given subcutaneously every 4-8 weeks, Tremfya’s success stems from its ability to inhibit the inflammatory protein cytokine IL-23. This protein escalates inflammation; therefore, Tremfya acts as an anti-inflammatory medicine.
This first-of-its-kind study is sponsored by Janssen Biotech, Inc., a division of Johnson & Johnson. Although it is no longer accepting new volunteers, another clinical trial that allows the possibility of being randomly assigned to receive Tremfya is being conducted through the IU School of Medicine and Riley Children's Health.
Tremfya has a way to go before being approved for pediatric use, but Pfefferkorn encourages families of patients with colitis whose bodies resist traditional treatment not to lose hope.
“There are several medications approved in adults and they're trickling into the pediatric population, and doctors are trained to treat and use the right medication for them.”
Pfefferkorn added it is imperative that families seek medical attention immediately if their children are experiencing long-term abdominal pain and diarrhea with blood in the stool.
“Blood is never normal in anyone's stool, so they need to be seen urgently,” Pfefferkorn said. “Colitis, just like other chronic diseases, for instance, diabetes, requires medication for life.”
With proper treatment and medication, a patient with colitis can live a healthy, normal life.
To contact the department of Pediatric Gastroenterology, Hepatology and Nutrition for additional questions, call: 317-944-3774 or visit: rileychildrens.org
