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

NCT ID: NCT06315738

Last Updated: 2025-05-22

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-08-19
• Study Completion Date: 2029-11-30

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.

Detailed Description

This Phase 1-2 clinical trial is a randomized, controlled, open-label study using a 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 ≥800 g and ≤999 g.

In each cohort, patients will be randomized to either ST266 + SOC or SOC alone. The first 3 patients randomized to ST266 will be staggered, where each patient must complete their 10 day treatment cycle and 1 month follow up visit and be evaluated by the Data Safety Monitoring Board (DSMB), before dosing of the next patient occurs. Patients randomized to SOC alone will follow the treatment plan as dictated by the Investigator or licensed medical designee and will be evaluated for the same inclusion/exclusion criteria and selected endpoints for analysis. If for any reason a patient is withdrawn, the decision for replacement will be determined by the DSMB.

Dosing for the next cohort will occur after review of safety data up to and including Day 28/1 Month from all patients in the previous cohort. The DSMB reviews will include comprehensive safety data analysis of data available at that time.

Conditions

Necrotizing Enterocolitis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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)

Group Type: OTHER

ST266

Intervention Type: 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 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)

Group Type: OTHER

ST266

Intervention Type: 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 3 - lower dose active + SOC treatment vs. SOC alone in lower weight range

Infants with weight at diagnosis of NEC ≥800 g and ≤999 g; 0.5 mL/kg of ST266, QD; + Standard of Care (SOC) treatment (n=6); SOC (n=3)

Group Type: OTHER

ST266

Intervention Type: 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 4 - higher dose active + SOC treatment vs. SOC alone in lower weight range

Infants with weight at diagnosis of NEC ≥800 g and ≤999 g; 1.0 mL/kg of ST266, QD; + Standard of Care (SOC) treatment (n=6); SOC (n=3)

Group Type: OTHER

ST266

Intervention Type: 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.

Interventions

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.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Infants born from ≥26 weeks gestational age to 40 weeks gestational age; up to 40 weeks postmenstrual gestational age (gestational age plus chronological age in terms of weeks) with current weight at diagnosis of NEC between ≥800g 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. Minimum Bell stage IIA NEC diagnosis by radiologic confirmed pneumatosis intestinalis and may include intestinal dilation and ileus.

Exclusion Criteria

1. Infants with abdominal perforation at less than 10 days of life

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.

• Minimum Eligible Age: 2 Weeks
• Maximum Eligible Age: 8 Weeks
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Parexel

INDUSTRY

Sponsor Role: collaborator

Noveome Biotherapeutics, formerly Stemnion

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Yale-New Haven Hospital

New Haven, Connecticut, United States

Site Status: RECRUITING

Orlando Health, Inc. Winnie Palmer Hospital for Women and Babies

Orlando, Florida, United States

Site Status: ACTIVE_NOT_RECRUITING

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

Tampa, Florida, United States

Site Status: RECRUITING

NorthShore University-Evanston Hospital

Evanston, Illinois, United States

Site Status: RECRUITING

Duke University Medical Center (DUMC)

Durham, North Carolina, United States

Site Status: ACTIVE_NOT_RECRUITING

Oklahoma Children's Hospital

Oklahoma City, Oklahoma, United States

Site Status: RECRUITING

University of Pittsburgh Medical Center Magee Womens Hospital

Pittsburgh, Pennsylvania, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Karin Potoka, MD

Role: CONTACT

kpotoka@noveome.com

412-512-1446

Shawna M Rose, BS

Role: CONTACT

srose@noveome.com

513-205-1091

Facility Contacts

Sarah Taylor, MD

Role: primary

sarah.n.taylor@yale.edu

203-688-2320

Jenelle Ferry, MD

Role: primary

jenelle.ferry@baycare.org

813-872-2924

Brandy Frost, MD

Role: primary

bfrost@northshore.org

773-562-7420

Hala Chaaban, MD

Role: primary

hala-chaaban@ouhsc.edu

405-271-5215 ext. 42063

Toby Yanowitz, MD

Role: primary

yanotd@upmc.edu

412-641-6260

Other Identifiers

ST266-NEC-201

Identifier Type: -

Identifier Source: org_study_id