Assessing ST266 for treating infants with Necrotizing Enterocolitis

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)

This study is testing if a new treatment called ST266 can help infants with Necrotizing Enterocolitis feel better when given along with their usual care.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 2 Weeks to 8 Weeks
• Sex: All
• Sponsor: Noveome Biotherapeutics, formerly Stemnion (industry)
• Locations: 8 sites (Little Rock, Arkansas and 7 other locations)
• Trial ID: NCT06315738 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and tolerability of two different doses of ST266 administered intravenously in infants diagnosed with Necrotizing Enterocolitis (NEC) at Bell's stage IIA or higher. The study employs a randomized, controlled, open-label design with a sequential cohort approach, where infants are assigned to receive either ST266 combined with standard of care or standard of care alone. The trial monitors treatment emergent adverse events and assesses preliminary efficacy over a treatment cycle of 10 days, followed by a one-month follow-up. The Data Safety Monitoring Board will oversee patient safety and dosing adjustments based on cohort evaluations.

Who should consider this trial

Good fit: Ideal candidates are infants born between 26 to 40 weeks gestational age, weighing between 800g and 3000g, diagnosed with Bell stage IIA or higher NEC.

Not a fit: Patients with abdominal perforation, those not expected to survive for at least two weeks, or those with major congenital anomalies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve outcomes for infants suffering from Necrotizing Enterocolitis, potentially reducing morbidity and mortality associated with this condition.

How similar studies have performed: While this approach is novel in the context of ST266 for NEC, similar studies have explored treatments for NEC with varying degrees of success.

Eligibility criteria

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.

Where this trial is running

Little Rock, Arkansas and 7 other locations

• Arkansas Children's Hospital — Little Rock, Arkansas, United States (RECRUITING)
• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States (RECRUITING)
• Yale-New Haven Hospital — New Haven, Connecticut, United States (RECRUITING)
• BayCare Health System-St. Joseph's Women's Hospital — Tampa, Florida, United States (RECRUITING)
• NorthShore University-Evanston Hospital — Evanston, Illinois, United States (RECRUITING)
• Oklahoma Children's Hospital — Oklahoma City, Oklahoma, United States (RECRUITING)
• Penn State Health Milton S Hershey Medical Center/Penn State University College of Medicine — Hershey, Pennsylvania, United States (RECRUITING)
• University of Pittsburgh Medical Center Magee Womens Hospital — Pittsburgh, Pennsylvania, United States (RECRUITING)

Study contacts

• Study coordinator: Karin Potoka, MD

Conditions: Necrotizing Enterocolitis, NEC