First In Human Study on Synthetic Surfactant CHF 5633 in Respiratory Distress Syndrome

Phase 1

Completed

• Conditions: Respiratory Distress Syndrome in Premature Infants
• Interventions: Drug: synthetic surfactant (CHF5633)

• Registration Number: NCT01651637
• Lead Sponsor: Chiesi Farmaceutici S.p.A.

Brief Summary

The aim of this study is to investigate the safety and tolerability of intratracheal administration of two different single doses of CHF 5633 in preterm neonates with RDS in terms of adverse events, adverse drug reactions, hematology and biochemistry values, the incidence of major neonatal morbidities including bronchopulmonary dysplasia (BPD) and mortality.

Detailed Description

The study will be a multicentre, open-label, single escalating dose, per-cohort design.

Study Timeline

• Study First Submitted: 2012-04-20
• Study First Submitted QC: 2012-07-26
• Study First Posted: 2012-07-27
• Study Start: 2012-10
• Primary Completion: 2015-01-23
• Study Completion: 2015-01-23
• Status Verified: 2020-07
• Last Update Submitted: 2020-07-30
• Last Update Posted: 2020-07-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Written informed consent obtained by parents/legal representative (according to local regulation) prior to any study-related procedures
• Inborn and outborn, preterm neonates of either sex with a gestational age of 27 weeks up to 33 weeks+6
• Clinical and radiological findings typical of RDS
• Age on admission to the study < 24 hours from birth
• Requirement of endotracheal intubation for surfactant administration
• Fraction of inspired oxygen (FiO2) > 0.35 to maintain SpO2 between 90-95 %
• Documentation of normal cranial ultrasound scan

Exclusion Criteria

• Use of surfactant prior to study entry and need for intratracheal administration of any other treatment (e.g. nitric oxide)
• Known genetic or chromosomal disorders, major congenital anomalies (cardiac malformations, myelomeningocele etc)
• Maternal drug abuse (heroin, methadone, methamphetamine, or cocaine) or significant alcohol consumption during pregnancy
• Clinical chorioamnionitis (Appendix III)
• Strong suspicion of congenital pneumonia/infection, sepsis
• Evidence of severe birth asphyxia or a 5 minutes Apgar score less / equal 3
• Presence of air leaks prior to study entry
• Neonatal seizures prior to study entry
• Mothers with prolonged rupture of the membranes (> 3 weeks duration)
• Any condition that, in the opinion of the Investigator, would place the neonate at undue risk
• Participation in another clinical trial of any placebo, drug or biological substance conducted under the provisions of a protocol

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Synthetic Surfactant	synthetic surfactant (CHF5633)	Cohort Design

Primary Outcome Measures

Name	Time	Method
Monitoring of adverse events following CHF5633 administration	For duration of hospital stay (expected average of 9 weeks)

Secondary Outcome Measures

Name	Time	Method
CHF 5633 efficacy profile (oxygenation , ventilatory requirements and need for rescue surfactant treatment),systemic absorption and immunogenicity assesment	At min 30, at hrs 1, 3, 6, 12, 24, at days 2, 3, 7 and at days 10, 28, at 36 weeks pma post dosing for ventilatory requirements, at pre-dose and 3 hrs and 24 hrs post dosing for systemic absorption and at 8 wks after administration for immunogenicity

Trial Locations

Locations (1)

Liverpool Women's Hospital Neonatal Unit Liverpool Women's Hospital; 🇬🇧 Liverpool, United Kingdom