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

NCT02452476 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: CCD-05633AA1-02
• Study Type: interventional
• Target: 123
• Locations: 1 country
• Sites: 23

Brief Summary

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%.

Conditions

Respiratory Distress Syndrome, Newborn

Keywords

neonates, pulmonary surfactant

Outcome Measures

Primary Outcomes (3)

• 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.

  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;

  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;

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

Other Outcomes (5)

• Number of Patients With Normal Breathing (Room Air) Within 24 Hours

  Post-treatment up to 24 h

• Number of Patients With the Need for Re-dosing (Use of Rescue Surfactant)

  Day 1 to Day 7

• Time to Reach Normal Breathing (Room Air) Within 24 Hours

  Post-treatment Day 1: up to 24 h

Study Arms (2)

CHF5633

EXPERIMENTAL

Single dose within 24 hours from birth

Drug: CHF5633

Poractant alfa

ACTIVE COMPARATOR

Single dose within 24 hours from birth

Drug: Poractant alfa

Interventions

CHF5633 DRUG

Rescue treatment (if needed)

CHF5633

Poractant alfa DRUG

Rescue treatment (if needed)

Poractant alfa

Eligibility Criteria

• Age: 1 Hour - 24 Hours
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Written informed consent obtained by parents/legal representative (according to local regulation) prior to any study-related procedures
• 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 SpO2 between 88-95%

You may not qualify if:

• 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
• Mothers with prolonged rupture of the membranes (\>21 days duration)
• Strong suspicion of congenital pneumonia/infection, sepsis
• Presence of air leaks prior to study entry
• Evidence of severe birth asphyxia
• Neonatal seizures prior to study entry
• 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.

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead

Study Sites (23)

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

Mobile, Alabama, United States

Location

LAC + USC Medical Center, Keck School of Medicine

Los Angeles, California, United States

Location

UC Irvine Medical Center

Orange, California, United States

Location

Sharp Mary Birch Hospital

San Diego, California, United States

Location

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Location

Plantation General Hospital (Sheridan Clinical Research, Inc.)

Plantation, Florida, United States

Location

Jatinder Bhatia

Augusta, Georgia, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, United States

Location

Memorial Hospital of South Bend

South Bend, Indiana, United States

Location

University of Louisville Research Foundation, Inc.

Louisville, Kentucky, United States

Location

Floating Hospital for Children at Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Baystate Children's Hospital / Baystate Medical Center

Springfield, Massachusetts, United States

Location

Winthrop University Hospital

Mineola, New York, United States

Location

Kings County Hospital Center

New York, New York, United States

Location

Sergio G. Golombek

Valhalla, New York, United States

Location

Martha Naylor

Greenville, North Carolina, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Location

Case Western Reserve University

Cleveland, Ohio, United States

Location

Krishnamurthy Sekar

Oklahoma City, Oklahoma, United States

Location

Hahnemann University Hospital

Philadelphia, Pennsylvania, United States

Location

Texas Tech University Health Sciences Center

El Paso, Texas, United States

Location

MultiCare Institute for Research & Innovation

Tacoma, Washington, United States

Location

West Virginia University

Morgantown, West Virginia, United States

Location

Related Publications (1)

• Ramanathan R, Biniwale M, Sekar K, Hanna N, Golombek S, Bhatia J, Naylor M, Fabbri L, Varoli G, Santoro D, Del Buono D, Piccinno A, Dammann CE. Synthetic Surfactant CHF5633 Compared with Poractant Alfa in the Treatment of Neonatal Respiratory Distress Syndrome: A Multicenter, Double-Blind, Randomized, Controlled Clinical Trial. J Pediatr. 2020 Oct;225:90-96.e1. doi: 10.1016/j.jpeds.2020.06.024. Epub 2020 Jun 14.

  PMID: 32553868 RESULT

MeSH Terms

Conditions

Respiratory Distress Syndrome, Newborn

Interventions

CHF5633; poractant alfa

Condition Hierarchy (Ancestors)

Respiratory Distress Syndrome; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Clinical Trial Transparency
• Organization: Chiesi Farmaceutici S.p.A.

clinicaltrials_info@chiesi.com

+39 0521 2791

Study Officials

• Rangasamy Ramanathan, MD

  Division of Neonatology, Department of Pediatrics, LAC+USC Medical Center and Good Samaritan Hospital, Keck School of Medicine of USC, Los Angeles, CA, USA

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 20, 2015
• First Posted: May 22, 2015
• Study Start: January 21, 2016
• Primary Completion: May 24, 2018
• Study Completion: May 24, 2018
• Last Updated: August 13, 2021
• Results First Posted: June 23, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (23)