Inflammatory Bowel Disease Treatment and Research Center | Current Research

The investigators at the Inflammatory Bowel Disease Treatment and Research Center continue to gather valuable data on the basis of Crohn’s disease and ulcerative colitis and their treatments through a variety of basic research and clinical trials. The following research initiatives are currently taking place at the Center and enrolling patients.

To enroll in a study, please talk to your gastroenterologist at your next appointment. If you have any specific questions, please contact the study coordinator assigned to each study below.

Very Early Onset Inflammatory Bowel Disease (VEOIBD) Consortium

Boston Children’s Hospital is the lead U.S. location for the Very Early Onset Inflammatory Bowel Disease (VEOIBD) Consortium (www.veoibd.org). This group of pediatric gastroenterologists and scientists from academic centers around the world are working together to identify the causes of VEOIBD with the goal of developing new therapeutic treatments for children with these rare diseases. Data gleaned from this initiative is helping us identify genetic mutations responsible for VEOIBD and define the molecular networks that are disrupted as a result of these mutations. We are also determining the environmental factors affecting genetic, microbial, and immunologic pathways relevant to VEOIBD and, based on these findings, developing tailored treatments for VEOIBD patients.

For more information, please contact: Caroline Rourke at 617-919-4592, or Caroline.Rourke@childrens.harvard.edu; or Spencer Evans at 617-919-4592, Spencer.Evans@childrens.harvard.edu, or Leslie Grushkin-Lerner at 617-919-8256 or Leslie.Gushkin-Lerner@childrens.harvard.edu.

Fecal Microbiota Transplant (FMT) for the Treatment of Pediatric Active Ulcerative Colitis

This phase I/II, double-blinded, placebo-controlled clinical trial is assessing the safety of FMT as a treatment for ulcerative colitis and Crohn’s disease. Patients will receive capsules of either FMT or placebo once a week for seven weeks, followed by eight weeks of open-label treatment for eligible patients.

For more information, please contact: : McKenzie Leier at 617-919-4609 or McKenzie.Leier@childrens.harvard.edu

A Randomized, Double-Blind Placebo Controlled Multi-Center Pragmatic Clinical Trial to Evaluate the Effectiveness of Low-Dose Oral Methotrexate in Patients with Pediatric Crohn’s Disease Initiating anti-TNF (COMBINE)

This double-blinded, placebo-controlled study is investigating the effects of combination therapy with the drugs infliximab and methotrexate. We hope to determine whether up to two years of combination therapy is better than a placebo in inducing and maintaining steroid-free remission from pediatric Crohn’s disease. We are currently enrolling Crohn’s disease patients who are younger than age 21 and starting an anti-TNF drug.

For more information, please contact: Knox Young, 857-218-4979 or Knox.Young@childrens.harvard.edu

Low Dose IL-2 for Ulcerative Colitis

IL-2, a cytokine produced by immune cells, when given at low doses, has been shown in preliminary studies to be effective in a number of immune-mediated conditions (e.g., graft versus host disease; SLE, vasculitis). Our goal is to learn whether low doses of IL-2 can also be used to treat ulcerative colitis through the proliferation of anti-inflammatory cells known as regulatory T-cells (T-regs). This study seeks to determine if eight weeks of daily treatment with IL-2 can promote the expansion of T-regs and lead to clinical improvement or remission in these patients. We are currently enrolling patients that are 18 years of age or older.

For more information, please contact: Caroline Rourke at 617-919-4592 or Caroline.Rourke@childrens.harvard.edu

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of the Human Anti-TNF Monoclonal Antibody Adalimumab in Pediatric Subjects with Moderate to Severe Ulcerative Colitis

We are conducting this industry-sponsored double-blinded, placebo-controlled trial to gain approval for the use of adalimumab (Humira) in pediatric ulcerative colitis patients. We are currently recruiting patients ages 4 to 17 with a diagnosis of ulcerative colitis.

For more information, please contact: Knox Young, 857-218-4979 or Knox.Young@childrens.harvard.edu

The Role of Viral Exposures Preceding IBD Diagnosis in Disease Pathogenesis

The virome—the collective name for all of the viruses a patient has ever contracted—is gaining more attention in IBD research. Viruses have been shown to have an important role in altering a patient’s immunity through interactions with other members of the microbiome, a patient’s intestinal lining and immune cells. Virscan is a newly developed technique that allows for a comprehensive look at all antiviral antibodies in a patient’s serum. Using this information, we are assessing the viromes of patients with Crohn's disease and ulcerative colitis both before and at diagnosis. This collaboration among Boston Children’s Hospital, Mt. Sinai Medical Center in NYC and the Naval Medical Research Center aims to identify specific viral triggers for IBD.

For more information, please contact: Tamar Parmet, 617-919-4592 or Tamar.Parmet@childrens.harvard.edu

Pediatric Gastrointestinal Disease Biospecimen Repository and Registry

We are collecting biological samples from patients with IBD, immunodeficiences or other GI disorders to create a one-of-a-kind bank of biospecimens and related clinical data. Our goal is to use this information to facilitate comprehensive studies on disease pathogenesis and progression. The biospecimen registry gives us the ability to ask specific questions about disease with the goal of identifying biomarkers that can predict disease outcome and response to therapy. In addition we are employing various next-generation sequence techniques including whole-exome sequencing and RNA-sequencing to identify genes and altered gene-networks that are responsible for disease initiation.

For more information, please contact: Caroline Rourke, 617-919-4592, or Caroline.Rourke@childrens.harvard.edu

The Hubble Study: A Phase 2, Dose-Ranging Study to Determine the Pharmacokinetics, Safety and Tolerability of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn’s Disease

This is a double-blind industry sponsored trial to determine the pharmacokinetics and safety of intravenous vedolizumab in pediatric subjects with Crohns disease or ulcerative colitis. We are recruiting subjects 2 to 17 years old with moderate to severe disease. Patients will be randomized to receive high or low dose vedolizumab. Subjects who complete the trial and are judged to have benefited from study drug will be eligible to receive vedolizumab in a 5 year extension study.

For more information, please contact: Knox Young, 857-218-4979 or Knox.Young@childrens.harvard.edu

Microbiome Study (Longitudinal stool)

We are determining how microbes in the gut are associated with inflammation, as well as with different treatments and environmental factors. We are collecting stool samples from patients with IBD, immunodeficiencies or other GI disorders, as well as from their household family members, to track changes in the gut microbiome over time. We are also capturing information about disease activity, treatments, dietary and environmental factors at the time of the sample collection.

For more information, please contact: Sarah Wall, 617-919-6957 or Sarah.Wall@childrens.harvard.edu

A Phase 4, Multicenter, Open-label Study of Serum Infliximab Concentrations and Efficacy and Safety of Dose Escalation in Pediatric Patients with Inflammatory Bowel Disease

This industry-sponsored trial will test the efficacy and safety of dose escalation (from 5mg/kg every eight weeks to 10mg/kg every eight weeks) for the drug infliximab (Remicade) in patients with IBD. We are currently recruiting patients between ages 6 and 17 with a biopsy-confirmed diagnosis of Crohn’s disease or ulcerative colitis.

For more information, please contact: Knox Young, 857-218-4979 or Knox.Young@childrens.harvard.edu

Moving research from bench to bedside and back

A number of laboratories in the division of GI/Nutrition include a variety of talented interdisciplinary investigators that focus on gastrointestinal research. These laboratories are interested in the cellular and molecular mechanisms of mucosal immunity and host-microbial cross-regulation that promote immune homeostasis and gastrointestinal health. In an effort to understand human gastrointestinal inflammatory disorders, we have established a research pipeline that moves our basic research findings into novel translational models, which funnel into new clinical trials.