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

NCT06315738 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ST266-NEC-201
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 8

Brief 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.

Conditions

Necrotizing Enterocolitis

Keywords

Necrotizing Enterocolitis; NEC

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (4)

• Efficacy endpoint: Time to pneumatosis resolution

  From date of NEC diagnosis until resolved, up to 10 days

• Efficacy endpoint: Time to full enteral nutrition assessment

  From date of completion of antibiotics and/or IP treatment until full feeding tolerance reached, 3-5 days

• Efficacy endpoint: Incidence of abdominal surgical intervention

  Assessed from Day 1/Baseline visit through 24 months of age

• Efficacy endpoint: Change in Neonatal Sequential Organ Failure Assessment (nSOFA) score

  From Randomization/Day 1 through Day 10 of treatment period (10 days).

Other Outcomes (17)

• Exploratory endpoint: All cause mortality

  From Day1/Baseline through 24 months of age

• Exploratory endpoint: Length of stay in the NICU

  From Day1/Baseline until date released from NICU - through 24 months of age

• Exploratory endpoint: Change in weight over time

  From Day1/Baseline through 24 months of age

Study Arms (4)

Cohort 1 - lower dose active + SOC treatment vs. SOC alone in higher weight range

OTHER

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

Cohort 2 - higher dose active + SOC treatment vs. SOC alone in higher weight range

OTHER

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

Cohort 3 - lower dose active + SOC treatment vs. SOC alone in lower weight range

OTHER

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

Cohort 4 - higher dose active + SOC treatment vs. SOC alone in lower weight range

OTHER

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

Interventions

ST266 BIOLOGICAL

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.

Cohort 1 - lower dose active + SOC treatment vs. SOC alone in higher weight range; Cohort 2 - higher dose active + SOC treatment vs. SOC alone in higher weight range; Cohort 3 - lower dose active + SOC treatment vs. SOC alone in lower weight range; Cohort 4 - higher dose active + SOC treatment vs. SOC alone in lower weight range

Eligibility Criteria

• Age: 2 Weeks - 8 Weeks
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• Infants with abdominal perforation.
• 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).
• Born with major congenital anomalies such as cardiac defects (e.g., Tetralogy of Fallot) or chromosomal disorders/anomalies (e.g., neural tube defect).
• Mother's receipt of any investigational product during pregnancy.
• Infants with malignancies (e.g., neoplastic cell growth as a solid tumor or a blood neoplasm, such as congenital leukemia).
• Infants with hypercoagulability disorders (any active thrombosis, diagnosis of disseminated intravascular coagulation or other acquired/inherited disorders (i.e., hemophilia) of coagulation.
• Infants with a known immunodeficiency (such as galactosemia or agranulocytosis).
• Infants with anatomic defects that require surgical intervention.
• Infants with persistent pulmonary hypertension of newborn.
• Infants with any congenital or acquired gastrointestinal pathology that preclude feeds within 7 days after birth (e.g., duodenal atresia).
• Infants who have hypoxic ischemic injury (perinatal asphyxia).
• Infants with polycythemia (at time of treatment) (\>22 g/dL).
• Positive maternal human immunodeficiency virus status.
• History of maternal drug abuse (such as amphetamines, opiates, cocaine). This does not include marijuana, or prescription medications for treatment of drug abuse.
• Considered by the Investigator, for any reason, to be an unsuitable candidate for the study.

Sponsors & Collaborators

• Noveome Biotherapeutics, formerly Stemnion: lead
• Parexel: collaborator

Study Sites (8)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

RECRUITING

Yale-New Haven Hospital

New Haven, Connecticut, 06510, United States

RECRUITING

BayCare Health System-St. Joseph's Women's Hospital

Tampa, Florida, 33607, United States

RECRUITING

NorthShore University-Evanston Hospital

Evanston, Illinois, 60201, United States

RECRUITING

Oklahoma Children's Hospital

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

Penn State Health Milton S Hershey Medical Center/Penn State University College of Medicine

Hershey, Pennsylvania, 17033, United States

RECRUITING

University of Pittsburgh Medical Center Magee Womens Hospital

Pittsburgh, Pennsylvania, 15219, United States

RECRUITING

Related Links

• Study Sponsor, Noveome Biotherapeutics, Inc., website

MeSH Terms

Conditions

Enterocolitis, Necrotizing

Condition Hierarchy (Ancestors)

Enterocolitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Central Study Contacts

Karin Potoka, MD

CONTACT

412-512-1446; kpotoka@noveome.com

Shawna M Rose, BS

CONTACT

513-205-1091; srose@noveome.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2024
• First Posted: March 18, 2024
• Study Start: August 19, 2024
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): November 1, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)