Because there is no approved front-line therapy for pediatric Crohn’s disease, significant discussion was undertaken with FDA to identify an acceptable and feasible adaptive Phase 3 clinical trial design. It is anticipated that only one trial of this design can be run in children, since once efficacy is demonstrated it would be unethical to run another controlled trial.
The SGX203 Phase 3 trial is ready to proceed pending partnership or funding.
Interested in partnering with us for this program?
Phase 3 Trial Design
The Phase 3 trial is a double-blind, randomized, controlled, multi-national trial that will enroll approximately 150 subjects, 6-17 years of age, with endoscopically proven mild to moderate Crohn’s disease. The trial will compare the rates of improvement of the signs and symptoms of Crohn’s disease after 8 weeks of treatment among subjects randomized to one of three SGX203 dose groups (split 60:30:60 among the lowest, middle and highest dose of SGX203). Subjects will be followed for an additional 6 months after the completion of treatment.
Entry criteria for the trial are the presence of Crohn’s disease symptoms (abdominal pain and/or diarrhea) associated with laboratory blood tests showing evidence of a disease flare.
The primary clinical efficacy endpoint of the trial will compare the percentage of subjects in each of the three dose groups having resolved their signs and symptoms after an 8-week course of treatment.
An adaptive design will be employed in which an independent Data Monitoring Committee will review the efficacy data after approximately 90 subjects have completed treatment and determine if the trial size requires adjustment based on the actual event rate, halted for futility, or overwhelming efficacy.
Previous Clinical Studies with SGX203
The safety of SGX203 was assessed in a Phase 1 study in 24 male and female healthy adolescents and young adults. SGX203 was found to be safe, to have no effect on bone metabolism (a common problem with systemic steroids) and to have limited systemic availability.
Other Potential Uses for BDP
A topical oral anti-inflammatory with action limited to the gastrointestinal tract may be useful in a number of other inflammatory conditions, including inflammation occurring as the result of radiation exposure.
Acute Radiation Syndrome (OrbeShield)
The SGX203 formulation is also being explored in the context of Acute Radiation Syndrome, as a potential therapy in the case of a mass-casualty nuclear accident or attack.
Inflammation of the gut after radiation therapy is also a common occurrence in the context of cancer treatment. In particular, radiation enteritis is a known side-effect of cancer treatment of bladder, uterus, cervix, rectum, prostate and vagina and in all cases, anti-inflammatory treatment of the gut lining may reduce the damage to the gut.
Symptoms of acute radiation enteritis include nausea, vomiting, abdominal pain and rectal bleeding. While the symptoms of most patients may fade a few weeks or months after radiation treatment, 20% of patients may go on to develop chronic radiation enteritis. There are over 100,000 patients annually in the US receiving abdominal or pelvic radiation treatment for cancer who are at risk of developing acute and chronic radiation enteritis. Oral BDP consists of the delayed release tablet containing BDP, specifically targeting the lower gastrointestinal tract.