Study to Assess the Safety, Tolerability, and Preliminary Efficacy of ST266 in Infants With Necrotizing Enterocolitis (NEC)
Start Date
8/19/2024
Completion Date
11/1/2029
Summary
The primary objective of this study is to determine the safety and tolerability of two dose levels (0.5 mL/kg and 1.0 mL/kg) of once daily (QD) via IV route of administration of ST266 in treating patients with Bell's stage IIA or higher medical NEC by incidence of treatment emergent adverse events (TEAEs) and SAEs, with a secondary objective to assess preliminary efficacy of the same two dose levels (0.5 mL/kg and 1.0 mL/kg) of QD via IV route of administration of ST266 in treating patients with Bell's stage IIA or higher medical NEC.
Detailed Description
This Phase 1-2 clinical trial is a randomized, controlled, open-label study using a modified sequential cohort design. Assignment to cohorts will be based on the following dosages and weight ranges: 0.5 mL/kg and 1.0 mL/kg; weight ≥1000 g and ≤3000 g, and weight ≥500 g and ≤999 g. In each cohort, patients will be randomized to either ST266 + SOC or SOC alone. In the first cohort, the first three patients randomized to ST266 were staggered, where each patient completed their 10-day treatment period containing 10 treatment cycles and Day 28/1 Month follow-up visit and were evaluated by the Data Safety Monitoring Board (DSMB), before dosing of the next patient occurred. Patients randomized to SOC alone followed the treatment plan as dictated by the Investigator site SOC procedures and were evaluated for the same inclusion/exclusion criteria and selected endpoints for analysis. If for any reason a patient was withdrawn, the decision for replacement was determined by the DSMB. Dosing for the next cohort will occur after review of safety data up to and including Day 28/1 Month post-treatment follow-up visit from all patients in Cohort 1. DSMB reviews will include comprehensive safety data analysis of data available at that time. In Cohorts 2, 3, and 4, only a single sentinel ST266-treated patient will be required to complete their 10-day treatment period containing 10 treatment cycles and Day 28/1 Month follow-up visit and be evaluated by the Data Safety Monitoring Board (DSMB), before dosing of the next patient occurs. Given that Cohort 2 shares the same weight range and Cohort 3 the same dose as Cohort 1, Cohorts 2 and 3 may be opened for enrollment in parallel. If any safety event occurs in either Cohort 2 or 3, the DSMB will promptly evaluate and determine whether to continue the study and/or reinstate patient staggering.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
ST266
Conditions
Locations
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas 72205
United States
Yale-New Haven Hospital
New Haven, Connecticut 06510
United States
BayCare Health System-St. Joseph's Women's Hospital
Tampa, Florida 33607
United States
NorthShore University-Evanston Hospital
Evanston, Illinois 60201
United States
Oklahoma Children's Hospital
Oklahoma City, Oklahoma 73104
United States
Penn State Health Milton S Hershey Medical Center/Penn State University College of Medicine
Hershey, Pennsylvania 17033
United States
University of Pittsburgh Medical Center Magee Womens Hospital
Pittsburgh, Pennsylvania 15219
United States