A Double Blind, Randomized, Controlled Study to Evaluate CHF 5633 (Synthetic Surfactant) and Poractant Alfa in Neonates With Respiratory Distress Syndrome (RDS) (POC)

Chiesi Farmaceutici S.p.A.123 enrolled

Overview

A multicenter, double blind, randomized, single dose, active-controlled study to investigate the efficacy and safety of synthetic surfactant (CHF 5633) in comparison to porcine surfactant (Poractant alfa, Curosurf ®) in the treatment of preterm neonates with respiratory distress syndrome. Main objectives of this study are to investigate the short term efficacy profile of CHF 5633 vs. porcine surfactant (Poractant Alfa, Curosurf®) in terms of reduced oxygen requirement and ventilatory support and to evaluate the mid-term efficacy profile in terms of reduced incidence of bronchopulmonary dysplasia (BPD) and mortality/BPD rate at 36 weeks post menstrual age (PMA), mortality rate at 28 days and 36 weeks PMA, RDS-associated mortality through 14 days of age and other major co-morbidities of prematurity. Inclusion criteria are: Written parental informed consent, inborn preterm neonates of either sex with a gestational age of 24+0 weeks up to 29+6 weeks, clinical course consistent with RDS, requirement of endotracheal surfactant administration within 24 hours from birth, fraction of inspired oxygen (FiO2) ≥0.30 for babies 24+0 to 26+6 weeks and FiO2 ≥0.35 for babies 27+0 to 29+6 weeks to maintain arterial oxygen saturation by pulse oximetry (SpO2) between 88-95%.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

123

Conditions

Respiratory Distress Syndrome, Newborn

Eligibility

Sex: ALLMin age: 1 HourMax age: 24 Hours

Medical Language ↔ Plain English

Inclusion Criteria: 1. Written informed consent obtained by parents/legal representative (according to local regulation) prior to any study-related procedures 2. Inborn preterm neonates of either sex with a gestational age of 24+0 weeks up to 29+6 weeks 3. Clinical course consistent with RDS 4. Requirement of endotracheal surfactant administration within 24 hours from birth 5. Fraction of inspired oxygen (FiO2) ≥0.30 for babies 24+0 to 26+6 weeks and FiO2 ≥0.35 for babies 27+0 to 29+6 weeks to maintain SpO2 between 88-95% Exclusion Criteria: 1. Use of surfactant prior to study entry and need for intratracheal administration of any other treatment (e.g. nitric oxide) 2. Known genetic or chromosomal disorders, major congenital anomalies (cardiac malformations, myelomeningocele etc) 3. Maternal drug abuse (heroin, methadone, methamphetamine, or cocaine) or significant alcohol consumption during pregnancy 4. Mothers with prolonged rupture of the membranes (\>21 days duration) 5. Strong suspicion of congenital pneumonia/infection, sepsis 6. Presence of air leaks prior to study entry 7. Evidence of severe birth asphyxia 8. Neonatal seizures prior to study entry 9. Any condition that, in the opinion of the Investigator, would place the neonate at undue risk 10. Participation in another clinical trial of any placebo, drug or biological substance conducted under the provisions of a protocol.

Locations (23)

University of South Alabama - USA Children's and Women's Hospital

Mobile, Alabama, United States

LAC + USC Medical Center, Keck School of Medicine

Los Angeles, California, United States

UC Irvine Medical Center

Orange, California, United States

Sharp Mary Birch Hospital

San Diego, California, United States

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Plantation General Hospital (Sheridan Clinical Research, Inc.)

Plantation, Florida, United States

Jatinder Bhatia

Augusta, Georgia, United States

Indiana University School of Medicine

Indianapolis, Indiana, United States

Memorial Hospital of South Bend

South Bend, Indiana, United States

University of Louisville Research Foundation, Inc.

Louisville, Kentucky, United States

...and 13 more locations

Outcomes

Primary Outcomes

Oxygen Requirement and Ventilatory Support -- SpO2/FiO2 Ratio

SpO2/FiO2 ratio The oxygen requirement and ventilatory support were assessed through arterial oxygen saturation, measured by pulse oximetry (SpO2 \[%\]) and ventilator settings, by measuring fraction of inspired oxygen (FiO2\[%\]) and SpO2/FiO2. Results are shown as change from baseline, summarized at post-treatment timepoints. Definitions: SpO2=Arterial Oxygen saturation by pulse oximetry; FiO2=Fraction of inspired oxygen; Baseline=The last pre-dose measurement taken on Day -1.

Time frame: Post-treatment Day 1: 30 min, at 1h, 3h, 6h, 12h, 18h, 24 h; Day 2, 3, 5, and 7

Fraction of Inspired Oxygen (FiO2) (Percent) During the First 24 h and up to Day 7

Fraction of inspired oxygen (FiO2) (percent) during the first 24 h and up to Day 7. Fraction of inspired oxygen (FiO2 \[percent\]). Results are shown as change from baseline, summarized at post-treatment time points. Definitions: FiO2=Fraction of inspired oxygen (percent); Baseline=The last pre-dose measurement taken on Day -1;

Time frame: Post-treatment Day 1: 30 min, at 1h, 3h, 6h, 12h, 18h, 24 h; Day 2, 3, 5, and 7

Number of Patients With Bronchopulmonary Dysplasia and Mortality

Bronchopulmonary dysplasia and mortality. Results summarize the following items: Number of patients who died and the number of patients who had bronchopulmonary dysplasia (BPD) were assessed by treatment, at 36 weeks post menstrual age (PMA). Number of patients who died by Day 28 post-natal age (PNA). Number of patients with respiratory distress syndrome (RDS)-associated mortality by Day 14 post-natal age (PNA). Definitions: BPD=Bronchopulmonary dysplasia; Mortality/BPD incidence=The incidence of neonates dead within 36-week PMA or alive at 36-week PMA with a diagnosis of BPD; PMA=Post menstrual age; PNA=Post-natal age; RDS=Respiratory distress syndrome;

Time frame: 36 weeks post menstrual age, Day 14 Post-Natal Age, Day 28 Post-Natal Age

A Double Blind, Randomized, Controlled Study to Evaluate CHF 5633 (Synthetic Surfactant) and Poractant Alfa in Neonates With Respiratory Distress Syndrome (RDS) (POC)

Overview

Conditions

Interventions

Eligibility

Locations (23)

Outcomes

Primary Outcomes