9 Meters Biopharma announced the design of its Phase 3 clinical trial of vurolenatide for adults with short bowel syndrome, or SBS. The study design follows the company’s successful End-of-Phase 2 meeting and incorporates input received from the FDA. The Phase 3 study, called VIBRANT 2, is a randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy, safety, and tolerability of vurolenatide 50 mg administered subcutaneously every two weeks for 12 weeks in adults with SBS. This international clinical study is designed to enroll approximately 105 patients with SBS and will be conducted in up to 50 clinical investigative sites in North America and Europe. The study population will include both SBS patients who meet the current parenteral support dependence definition and SBS patients who do not meet this PS requirement threshold. Patients with SBS suffer from severe malabsorption due to the lack of sufficient intestinal surface which results directly in severe and often debilitating fluid and nutritional losses in the form of chronic, recurrent diarrhea. This study will not only assess the degree to which vurolenatide can reduce weekly PS volume requirements, but it will also, for the first time in a large ambulatory study, assess the impact of vurolenatide on malabsorptive diarrhea as measured directly by total stool output (TSO) volume. To maximize the potential for vurolenatide to provide clinical benefit to the entire SBS population regardless of PS requirement, VIBRANT 2 incorporates two primary efficacy endpoints: 1) change from baseline in weekly PS volume which was the established primary efficacy outcome measure to support the approval of the GLP-2 agonist Gattex for treatment of SBS patients with a PS dependence; and 2) change from baseline in mean 24-hour TSO volume, which assesses TSO volume over the entire treatment period and incorporates specific FDA recommendations around the inclusion of nutrition and hydration parameters to help establish the clinical relevance of this novel endpoint. Reduction in PS and TSO are both important clinical signs indicative of patients’ ability to absorb nutrients and fluids. It is planned that success on either primary efficacy endpoint can be the basis for a potential future New Drug Application submission for vurolenatide. In addition, an interim analysis is planned specifically for the PS-dependent patient population. When 50% of patients with PS dependence reach week 12, the interim analysis will determine whether an additional 12 weeks, which to date has been the regulatory predicate for GLP-2 agonists, will be required to assess safety and efficacy. All patients who complete the VIBRANT 2 double-blind treatment period will be eligible to enter an open-label extension study with the objective of assessing the long-term safety of vurolenatide for up to 12 months. U.S. Institutional Review Board approval has been secured, and study initiation is planned as early as the end of this year. Furthermore, several key features have been incorporated that we believe will optimize the ability to enroll the study, including utilizing a clinical research organization with specific experience executing late-stage clinical studies in SBS; securing up to 50 clinical study sites globally; and giving investigators the ability to enroll all SBS patients, regardless of PS status.
Published first on TheFly
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