Necrotizing Enterocolitis Clinical Trial
Official title:
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
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.
Status | Recruiting |
Enrollment | 36 |
Est. completion date | August 2027 |
Est. primary completion date | December 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Weeks to 8 Weeks |
Eligibility | Inclusion Criteria: 1. Premature (=26 and =36 weeks gestational age) infant weight between =800g and =3000g. 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. 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. 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. 15. Considered by the Investigator, for any reason, to be an unsuitable candidate for the study. |
Country | Name | City | State |
---|---|---|---|
United States | Duke University Medical Center (DUMC) | Durham | North Carolina |
United States | Orlando Health, Inc. Winnie Palmer Hospital for Women and Babies | Orlando | Florida |
United States | BayCare Health System-St. Joseph's Women's Hospital | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
Noveome Biotherapeutics, formerly Stemnion | Parexel |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Exploratory endpoint: All cause mortality | Assessment of all deaths that occurred during the study | From Day1/Baseline through 6 month follow up visit - up to 6 months | |
Other | 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 - up to 6 months | |
Other | Exploratory endpoint: Change in weight over time | Weight will be measured in grams (g) | From Day1/Baseline through 6 month follow-up visit - up to 6 months | |
Other | Exploratory endpoint: Change in length over time | Length will be measured in centimeters (cm) | From Day1/Baseline through 6 month follow-up visit - up to 6 months | |
Other | 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 6 month follow-up visit - up to 6 months | |
Other | 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 | |
Other | 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 | |
Other | 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 | |
Other | 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 | |
Other | 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 | |
Other | 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 | |
Other | 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 | |
Other | 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 | |
Other | Exploratory endpoint: Incidence of retinopathy | To be monitored while in NICU | From Day1/Baseline until resolved - up to 6 months | |
Other | 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 | |
Other | 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 | |
Other | 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 6 month post treatment follow up visit - up to 6 months | |
Primary | 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 6 month follow-up visit | |
Primary | 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 6 month follow-up visit | |
Primary | 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 6 month follow-up visit | |
Secondary | 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 | |
Secondary | 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 | |
Secondary | Efficacy endpoint: Incidence of abdominal surgical intervention | Abdominal surgical intervention defined as laparotomy, including drain placement | Assessed from Day 1/Baseline visit through 6 month follow up visit; up to 6 months | |
Secondary | 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). |
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