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

NCT ID: NCT02452476

Last Updated: 2021-08-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 123 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-01-21
• Study Completion Date: 2018-05-24

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

CHF5633

Single dose within 24 hours from birth

Group Type: EXPERIMENTAL

CHF5633

Intervention Type: DRUG

Rescue treatment (if needed)

Poractant alfa

Single dose within 24 hours from birth

Group Type: ACTIVE_COMPARATOR

Poractant alfa

Intervention Type: DRUG

Rescue treatment (if needed)

Interventions

CHF5633

Rescue treatment (if needed)

Intervention Type: DRUG

Poractant alfa

Rescue treatment (if needed)

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 1 Hour
• Maximum Eligible Age: 24 Hours
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Chiesi Farmaceutici S.p.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Rangasamy Ramanathan, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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

Floating Hospital for Children at Tufts Medical Center

Boston, Massachusetts, United States

Baystate Children's Hospital / Baystate Medical Center

Springfield, Massachusetts, United States

Winthrop University Hospital

Mineola, New York, United States

Kings County Hospital Center

New York, New York, United States

Sergio G. Golombek

Valhalla, New York, United States

Martha Naylor

Greenville, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Case Western Reserve University

Cleveland, Ohio, United States

Krishnamurthy Sekar

Oklahoma City, Oklahoma, United States

Hahnemann University Hospital

Philadelphia, Pennsylvania, United States

Texas Tech University Health Sciences Center

El Paso, Texas, United States

MultiCare Institute for Research & Innovation

Tacoma, Washington, United States

West Virginia University

Morgantown, West Virginia, United States

Countries

United States

References

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.

Reference Type: RESULT

PMID: 32553868 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CCD-05633AA1-02

Identifier Type: -

Identifier Source: org_study_id