Outcomes study for the XL1 distraction enterogenesis device to improve nutrient absorption in short bowel syndrome patients

PROJECT SUMMARY Short Bowel Syndrome (SBS) is a rare and devastating condition where patients are unable to absorb enough nutrients naturally to sustain life. SBS results when certain diseases or conditions that destroy the small intestine force the loss and surgical removal of the majority of the small intestine. In the pediatric patient population, the most common diseases that lead to SBS are necrotizing enterocolitis, midgut volvulus, and intestinal atresia. In the adult population, diseases include Crohn’s, cancer, intestinal ischemia, and trauma. Whatever its cause, SBS leaves the patient without enough functional small intestine to absorb nutrients from the food they consume. Current chronic therapies for SBS include parenteral (intravenous) nutrition, daily medication, and intestinal surgery. None of these treatments restore natural absorption of nutrients, and they are associated with significant complications and costs. Complications from daily parenteral nutrition include liver failure and central line infec- tions (sepsis), and mortality rates as high as 30% by age 5 have been reported. Current treatments are also staggeringly costly: the average 5-year cost has been estimated at $1.6M per patient. For pediatric patients, the costs of treatment can exceed $500,000 in the first year, and they may require treatment their entire lives. Hence, there is an urgent need for a novel treatment that can offer SBS patients a cure. Clinical studies have shown that for every additional 1% of residual small bowel a child possesses, the odds of weaning from parenteral nutrition increases by 3%. Over the last decade, intestinal lengthening, via a process called distraction enterogenesis––stretching the intestine to force it to grow, has emerged as a potential restor- ative treatment option that can help SBS patients grow new small intestine. Eclipse has been developing a novel mechanical device––the Eclipse XL1 System for Distraction Entero- genesis––that can stimulate the growth of healthy small intestine tissue. The XL1 device is currently in a phased safety study before Eclipse begins a probable benefit (HDE) study to support an application for approval as a Humanitarian Use Device. The proposed project will support the probable benefit phase of the study. It will enable Eclipse to treat patients at existing trial sites (Cincinnati Children's Hospital and Boston Children's Hospital; participated in the safety phase) to get to the projected halfway point of the probable benefit study in the first year. It will allow the trial to expand to two new study sites, giving the study access to distinct patient populations for a more inclusive dataset as well as a larger geographic footprint that should speed adoption once the treatment is approved. Finally, we will construct a retrospective control cohort of SBS cases that were medically managed; comparing outcomes for the treated cohort to those of controls will help demonstrate the benefit of the XL1 device. Thus, this study will directly support and accelerate Eclipse's efforts to secure XL1 regulatory approval via the HDE pathway, the final step in bringing this ground-breaking, curative treatment for SBS to patients.