Study to Assess the Safety, Tolerability, and Preliminary Efficacy of ST266 in Infants With Necrotizing Enterocolitis (NEC) (NEC)

Randomized, Controlled, Phase 1-2 Open Label Study of ST266 IV Administration to Assess the Safety, Tolerability, and Preliminary Efficacy of ST266 in Infants With Necrotizing Enterocolitis (NEC)

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.

Study Overview

• Status: Recruiting
• Conditions: Necrotizing Enterocolitis
• Intervention / Treatment: Biological: ST266

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 If for any reason a patient was withdrawn, the decision for replacement was determined by the DSMB.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.

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Karin Potoka, MD; Phone Number: 412-512-1446; Email: kpotoka@noveome.com
• Study Contact Backup: Name: Shawna M Rose, BS; Phone Number: 513-205-1091; Email: srose@noveome.com

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Recruiting
      • Arkansas Children's Hospital
      • Contact: Vikas Chowdhary, MD; Phone Number: 501-364-1028; Email: vchowdhary@uams.edu
      • Principal Investigator: Vikas Chowdhary, MD
      • Sub-Investigator: Sherry Courtney, MD
    • Little Rock, Arkansas, United States, 72205
      • Recruiting
      • University of Arkansas for Medical Sciences
      • Principal Investigator: Vikas Chowdhary, MD
      • Sub-Investigator: Sherry Courtney, MD
      • Contact: Vikas Chowdhary, MD; Phone Number: 848-702-3188; Email: vchowdhary@uams.edu
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Recruiting
      • Yale-New Haven Hospital
      • Contact: Sarah Taylor, MD; Phone Number: 203-688-2320; Email: sarah.n.taylor@yale.edu
      • Principal Investigator: Sarah Taylor, MD
      • Sub-Investigator: Kathryn Fletcher, MD
      • Sub-Investigator: Samuel Gentle, MD
      • Sub-Investigator: Catherine Buck, MD
  • Florida
    • Tampa, Florida, United States, 33607
      • Recruiting
      • BayCare Health System-St. Joseph's Women's Hospital
      • Contact: Jenelle Ferry, MD; Phone Number: 813-872-2924; Email: jenelle.ferry@baycare.org
      • Principal Investigator: Jenelle Ferry, MD
      • Sub-Investigator: Monisha Saste, MD
  • Illinois
    • Evanston, Illinois, United States, 60201
      • Recruiting
      • NorthShore University-Evanston Hospital
      • Contact: Brandy Frost, MD; Phone Number: 773-562-7420; Email: bfrost@northshore.org
      • Principal Investigator: Brandy Frost, MD
      • Sub-Investigator: Michael Caplan, MD
      • Sub-Investigator: Matthew Derrick, MD
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Recruiting
      • Oklahoma Children's Hospital
      • Contact: Hala Chaaban, MD; Phone Number: 42063 405-271-5215; Email: hala-chaaban@ouhsc.edu
      • Principal Investigator: Hala Chaaban, MD
      • Sub-Investigator: Pratibha Thakkar, MD
      • Sub-Investigator: Catherine Hunter, MD
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Recruiting
      • Penn State Health Milton S Hershey Medical Center/Penn State University College of Medicine
      • Contact: Timothy Palmer, MD; Phone Number: 717-531-8413; Email: tpalmer@pennstatehealth.psu.edu
      • Principal Investigator: Timothy Palmer, MD
      • Sub-Investigator: Shaili Amatya, MD
      • Sub-Investigator: Tammy Corr, DO
      • Sub-Investigator: Chintan Gandhi, MD
      • Sub-Investigator: Sarah Mahdally, DO
      • Sub-Investigator: Sara Mola, MD
    • Pittsburgh, Pennsylvania, United States, 15219
      • Recruiting
      • University of Pittsburgh Medical Center Magee Womens Hospital
      • Contact: Toby Yanowitz, MD; Phone Number: 412-641-6260; Email: yanotd@upmc.edu
      • Principal Investigator: Toby Yanowitz, MD
      • Sub-Investigator: Nicole Dobson, MD
      • Sub-Investigator: Brighid O'Donnell, MD
      • Sub-Investigator: Abeer Azzuqa, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Infants born from ≥22 weeks gestational age up to and including 40 weeks gestational age; up to 40 weeks postmenstrual age (gestational age plus chronological age in terms of weeks) with current weight at diagnosis of NEC between ≥500g and ≤3000g, as a result of prematurity and/or IUGR. Parent(s)/legal medical representative(s) voluntarily provides written consent prior to study enrollment.
2. Bell's Stage IIA or higher medical NEC (Stages IIA - IIIA only) diagnosis by radiologic confirmed pneumatosis intestinalis and may include intestinal dilation and ileus. The clinician confirms NEC diagnosis by evaluation of the radiologic imaging for confirmed pneumatosis intestinalis. If X-ray is used and is equivocal, an ultrasound (US) may be used, if available, to confirm pneumatosis. If the clinician (Neonatologist and/or Pediatric Surgeon) has differing interpretation from that of the Radiologist, that should be documented in both the medical and research records for accuracy of NEC diagnosis.

Exclusion Criteria:

1. Infants with abdominal perforation.
2. Not expected to survive ≥2 weeks or born with a lethal condition requiring hospice or palliative care (e.g., disease has progressed to NEC totalis, or patient has multi-organ system failure).
3. Born with major congenital anomalies such as cardiac defects (e.g., Tetralogy of Fallot) or chromosomal disorders/anomalies (e.g., neural tube defect).
4. Mother's receipt of any investigational product during pregnancy.
5. Infants with malignancies (e.g., neoplastic cell growth as a solid tumor or a blood neoplasm, such as congenital leukemia).
6. Infants with hypercoagulability disorders (any active thrombosis, diagnosis of disseminated intravascular coagulation or other acquired/inherited disorders (i.e., hemophilia) of coagulation.
7. Infants with a known immunodeficiency (such as galactosemia or agranulocytosis).
8. Infants with anatomic defects that require surgical intervention.
9. Infants with persistent pulmonary hypertension of newborn.
10. Infants with any congenital or acquired gastrointestinal pathology that preclude feeds within 7 days after birth (e.g., duodenal atresia).
11. Infants who have hypoxic ischemic injury (perinatal asphyxia).
12. Infants with polycythemia (at time of treatment) (>22 g/dL).
13. Positive maternal human immunodeficiency virus status.
14. History of maternal drug abuse (such as amphetamines, opiates, cocaine). This does not include marijuana, or prescription medications for treatment of drug abuse.
15. Considered by the Investigator, for any reason, to be an unsuitable candidate for the study.
16. Infants diagnosed with NEC who will require immediate surgical intervention.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Cohort 1 - lower dose active + SOC treatment vs. SOC alone in higher weight range; Infants with weight at diagnosis of NEC ≥1000 g and ≤3000 g; 0.5 mL/kg of ST266, QD, + Standard of Care (SOC) treatment (n=6); SOC (n=3)	Biological: ST266; Patients randomized to investigative drug product (ST266) will receive either 0.5 mL/kg or 1.0 mL/kg of ST266 QD in addition to Standard of Care treatment; Patients randomized to SOC will receive standard of care treatment only.
Other: Cohort 2 - higher dose active + SOC treatment vs. SOC alone in higher weight range; Infants with weight at diagnosis of NEC ≥1000 g and ≤3000 g; 1.0 mL/kg of ST266, QD; + Standard of Care (SOC) treatment (n=6); SOC (n=3)	Biological: ST266; Patients randomized to investigative drug product (ST266) will receive either 0.5 mL/kg or 1.0 mL/kg of ST266 QD in addition to Standard of Care treatment; Patients randomized to SOC will receive standard of care treatment only.
Other: Cohort 3 - lower dose active + SOC treatment vs. SOC alone in lower weight range; Infants with weight at diagnosis of NEC ≥500 g and ≤999 g; 0.5 mL/kg of ST266, QD; + Standard of Care (SOC) treatment (n=6); SOC (n=3)	Biological: ST266; Patients randomized to investigative drug product (ST266) will receive either 0.5 mL/kg or 1.0 mL/kg of ST266 QD in addition to Standard of Care treatment; Patients randomized to SOC will receive standard of care treatment only.
Other: Cohort 4 - higher dose active + SOC treatment vs. SOC alone in lower weight range; Infants with weight at diagnosis of NEC ≥500 g and ≤999 g; 1.0 mL/kg of ST266, QD; + Standard of Care (SOC) treatment (n=6); SOC (n=3)	Biological: ST266; Patients randomized to investigative drug product (ST266) will receive either 0.5 mL/kg or 1.0 mL/kg of ST266 QD in addition to Standard of Care treatment; Patients randomized to SOC will receive standard of care treatment only.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and Tolerability endpoint: incidence of adverse events	Patients will be assessed for safety and tolerability of ST266 treatment given IV (two dose options: 0.5mL/kg or 1.0mL/kg) via review of treatment emergent adverse events (TEAEs). AEs are defined as per the International Neonatal Consortium (INC) neonatal AE severity scale (NAESS): Mild, Moderate, Severe, Life Threatening, Death. Relatedness to study drug (ST266) will also be assessed.	From date of randomization through 24 months of age
Safety and Tolerability endpoint: incidence of serious adverse events	Patients will be assessed for safety and tolerability of ST266 treatment given IV (two dose options: 0.5mL/kg or 1.0mL/kg) via review of serious adverse events, defined as: any event that results in death, is immediately life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, or may jeopardize patient so that requires intervention to prevent prior noted outcomes (includes study drug related dose-limiting toxicities and infusion reactions)	From date of randomization through 24 months of age
Safety and Tolerability endpoint: Changes in labs and vitals relative to disease progression	Patients will be assessed for safety and tolerability of ST266 treatment given IV (two dose options: 0.5mL/kg or 1.0mL/kg) by evaluating clinically significant changes in labs and vitals as to relatedness to disease progression and study drug effects, including assessment of vital signs (temperature, systolic and Mean arterial blood pressure (mmHg), respiratory rate, heart rate, and percent oxygen saturation as measured by pulse oximetry as noted in The Harriet Lane Handbook, 21st ed., 2018), and hematology and chemistry lab tests, which will be measured against standard laboratory acceptable ranges for premature infants.	From date of randomization through 24 months of age

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy endpoint: Time to pneumatosis resolution	Pneumatosis is considered resolved when no longer observed on abdominal x-ray	From date of NEC diagnosis until resolved, up to 10 days
Efficacy endpoint: Time to full enteral nutrition assessment	Defined as no longer receiving total parenteral nutrition (TPN)	From date of completion of antibiotics and/or IP treatment until full feeding tolerance reached, 3-5 days
Efficacy endpoint: Change in Neonatal Sequential Organ Failure Assessment (nSOFA) score	nSOFA score is a neonatal sequential organ failure assessment of three organ systems: respiratory score criteria (range: 0-8); cardiovascular score criteria (range: 0-4); and Hematologic score criteria (range: 0-3) with a total score range: 0 (best) to 15 (worst)	From Randomization/Day 1 through Day 10 of treatment period (10 days).
Efficacy endpoint: Incidence of abdominal surgical intervention	Abdominal surgical intervention defined as laparotomy, including drain placement	Assessed from Day 1/Baseline visit through 24 months of age

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory endpoint: Number of significant apnea events/day over time	Significant apnea: any apnea for greater than 20 seconds	From Day1/Baseline through 1 month follow-up visit - up to 2 months
Exploratory endpoint: Number of significant bradycardia events/day over time	Significant bradycardia: any bradycardia heart rate < 70BPM for > 6 seconds	From Day1/Baseline through 1 month follow-up visit - up to 2 months
Exploratory endpoint: Number of significant temperature instability events/day over time	Significant temperature instability event: temperature < 36.5C or > 37.5C outside neutral/thermal environment or requires a change in isolette temperature outside of standard protocol	From Day1/Baseline through 1 month follow-up visit - up to 2 months
Exploratory endpoint: Time to return to normal bowel sounds	Defined as are typically soft and gurgling, indicating pneumatosis resolution and no feeding intolerance, no bowel obstruction or perforation.	From date of NEC diagnosis until resolved, up to 10 days
Exploratory endpoint: Change in serum C-reactive protein (CRP)	Included as part of labs being assessed per standard of care treatment	To be assessed on Day2 and Day8 during treatment period; and 2 weeks and 1 month post treatment
Exploratory endpoint: Time to return to normal serum platelet count levels	Included as part of labs being assessed per standard of care treatment	To be assessed on Day2 and Day8 during treatment period; and 2 weeks and 1 month post treatment
Exploratory endpoint: Incidence of intestinal or colonic strictures	Measured by fluoroscopic study (barium enema or upper gastrointestinal scope); only conducted if infant had symptoms indicating partial bowel obstruction (e.g., feeding intolerance, abdominal distension, bilious emesis).	From Day1/Baseline through Day28/1 month post treatment follow up visit
Exploratory endpoint: Incidence of sepsis	Defined as presence of a pathogenic bacteria in blood culture or two positive blood cultures	From Day1/Baseline through Day 28/1 month post treatment follow up visit
Exploratory endpoint: Incidence of bronchopulmonary dysplasia (BPD)	To be identified at 36 weeks CGA and at discharge from NICU	At 36 weeks of age and at discharge from NICU - up to 6 months
Exploratory endpoint: Incidence of pulmonary hypertension (PH)	To be identified at 36 weeks CGA and at discharge from NICU	At 36 weeks of age and at discharge from NICU - up to 6 months
Exploratory endpoint: All cause mortality	Assessment of all deaths that occurred during the study	From Day1/Baseline through 24 months of age
Exploratory endpoint: Length of stay in the NICU	Defined as total number of days from Day1/Baseline until date released from NICU	From Day1/Baseline until date released from NICU - through 24 months of age
Exploratory endpoint: Change in weight over time	Weight will be measured in grams (g)	From Day1/Baseline through 24 months of age
Exploratory endpoint: Change in length over time	Length will be measured in centimeters (cm)	From Day1/Baseline through 24 months of age
Exploratory endpoint: Change in head circumference over time	Circumference will be measured in centimeters (cm) and will be used to ensure normal head growth.	From Day1/Baseline through 24 months of age
Exploratory endpoint: Incidence of retinopathy	To be monitored while in NICU	From Day1/Baseline until resolved - through 24 months of age
Exploratory endpoint: Incidence of periventricular leukomalacia	To be measured via any form of imaging and noted if any symptoms observed	From Day 1 post treatment follow up visit through 24 months of age

Collaborators and Investigators

• Sponsor: Noveome Biotherapeutics, formerly Stemnion
• Collaborators: Parexel

Publications and helpful links

Helpful Links

• Study Sponsor, Noveome Biotherapeutics, Inc., website

Study record dates

Study Major Dates

• Study Start (Actual): August 19, 2024
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): November 1, 2029

Study Registration Dates

• First Submitted: March 1, 2024
• First Submitted That Met QC Criteria: March 13, 2024
• First Posted (Actual): March 18, 2024

Study Record Updates

• Last Update Posted (Actual): April 13, 2026
• Last Update Submitted That Met QC Criteria: April 7, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: NEC; Necrotizing Enterocolitis
• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Enterocolitis; Enterocolitis, Necrotizing
• Other Study ID Numbers: ST266-NEC-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No