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

Chiesi

Status and phase: Completed; Phase 2

Conditions: Respiratory Distress Syndrome, Newborn

Treatments: Drug: Poractant alfa; Drug: CHF5633

Study type: Interventional

Funder types: Industry

Identifiers

NCT02452476

CCD-05633AA1-02

Details and patient eligibility

About

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%.

Enrollment: 123 patients

Sex: All

Ages: 1 to 24 hours old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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123 participants in 2 patient groups

CHF5633

Experimental group

Description:

Single dose within 24 hours from birth

Treatment:

Drug: CHF5633

Poractant alfa

Active Comparator group

Description:

Single dose within 24 hours from birth

Treatment:

Drug: Poractant alfa