A phase II study of enterade in neuroendocrine tumor (NET) patients with quality-of-life limiting bowel movement frequency.

Abstract

TPS634 Background: BM frequency in NET pts is a major cause of morbidity. Though typically associated with serotonin excess in pts with carcinoid syndrome (CS), it can also occur in pts without CS due to etiologies such as short bowel syndrome, bile acid malabsorption, steatorrhea from somatostatin analogs (SSAs) and complications from chemotherapy/immunotherapy/biologics. Enterade is an oral rehydration solution, comprised of 5 amino acids, which has demonstrated pre-clinical and clinical potential to improve small intestinal resorption, thereby offering a potential treatment for limiting BM frequency in NET pts. Methods: We are conducting a phase II parallel cohort study in CS and non-CS NET pts (NCT 4073017). To be eligible, pts must be experiencing an average of ≥ 4 daily BM while on standard-of-care (SOC) therapies (SSAs, anti-diarrheals). Pts will be categorized into CS or non-CS cohorts based upon biochemical evaluation which includes plasma 5-HIAA/24-hour urine 5-HIAA, VIP or gastrin levels. Eligible pts will undergo observation during weeks 1-4 (baseline period). During weeks 5-8, pts will consume Enterade twice per day (Enterade period) while during weeks 9-12, pts will stop Enterade and resume observation. Daily stool diaries for each pt will be assessed to compute differences in mean bowel movement number between baseline and Enterade periods. Endpoints: The primary endpoint of the study is reduction in BM frequency in individual pts between baseline and Enterade periods. Based on parameters utilized in a prior study, we will assume that the mean daily reduction in bowel movements from baseline is equal to 1.5 (standard deviation of change = 1.5) representing a large effect size = 1.0. A sample of 12 pts in each cohort will provide nearly 90% power to detect this effect size based on a one-sample t-test (α = .05). An additional 3 pts will be added to each cohort to account for potential dropouts. Key secondary study endpoints include differences in pt-reported QOL outcomes (FACIT-D), pt weight, intravenous fluid requirement and utilization of SOC anti-diarrheals between baseline and Enterade periods. Accrual for the study will be ensuing shortly. Clinical trial information: NCT04073017.