The purpose of this research study is to determine which dose is effective and how the body processes the study drug, vedolizumab in children ages 2-17 who have ulcerative colitis or Crohn's disease.
This phase 2 study includes a 4-week Screening Period, a 22-week
Treatment Period (with last dose at Week 14). Subjects who do not
enter the long term extension study will have an 18-week Follow-up Period starting
from their last dose of study drug and a long-term follow-up safety
survey by telephone 6 months after their last dose of study drug.
This is a long-term extension study enrolling male and female pediatric subjects with ulcerative colitis (UC) or Crohn's disease (CD) who initiated vedolizumab intravenous (IV) treatment in the phase 2 Study MLN0002-2003 between the ages of 2 and 17 years of age. The study will evaluate the long-term safety of vedolizumab administered by IV infusion in pediatric subjects with UC or CD. The study will also evaluate the effect of long-term vedolizumab IV treatment on the time to major IBD-related events (hospitalizations, surgeries, or procedures), health-related quality-of-life measurements, patterns of growth and development, and exploratory efficacy measures. Up to 80 rollover subjects from Study MLN0002-2003 will participate in this study. Subjects who weigh 30 kg or greater will receive vedolizumab 300 mg (high dose) or 150 mg (low dose) Q8W. Subjects who weight less than 30 kg will receive vedolizumab 200 mg (high dose) or 100 mg (low dose) IV. Patients will receive treatment and perform assessments every 8 weeks for up to 5 years or until the subject withdraws from the study or the sponsor decides to close the study, whichever comes first.
A Crohn's Disease Registry for pediatric patients (between the ages of 6 and 17 years) with moderately to severely active Crohn's Disease (CD), who are already taking Humira® (Adalimumab) to treat their condition. Data from the participant's medical record will be collected to study the long-term safety and effectiveness of Humira®. The Registry expects to collect data for 10 years. No study drug will be given.
Hirschsprung disease (HSCR) is a birth defect resulting from the absence of nerve (ganglion) cells in the gastrointestinal tract. Hirschsprung disease has a population incidence of 1/5000 live births and most often occurs as an isolated condition. However, approximately 30% of HSCR cases are associated with other birth defects such as Down syndrome, deafness, hypopigmentation, and Ondine's curse (Congenital Central Hypoventilation syndrome (CCHS)). Hirschsprung disease is a genetic condition with autosomal dominant, autosomal recessive, and multigenic patterns of inheritance described. While several genes associated with HSCR have been identified, it is expected that additional genes play a role in the disorder. Furthermore, much remains to be understood about the mechanisms of genetic variants involved in the disease and how variants in multiple genes interact to lead to multiigenic forms of HSCR.
The objective of the Hirschsprung Disease Research Collaborative (HDRC) is to build a large collection of data and biological samples of individuals with HSCR by which genetic data can be linked to detailed and accurate phenotypic information about study participants. The goal of the genetic studies carried out with HDRC samples is to identify genes harboring causative HSCR mutations and to better understand the complex inheritance of HSCR in families by whole genome mapping and sequencing studies. Specifically, we intend to ascertain the frequency with which mutations in any human gene lead to familial and isolated forms of HSCR. Further, clinical information will be used to investigate possible genotype ? phenotype correlations and their relationship with medical, surgical and pathological data on patients.