Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Premature Neonates

A Multicenter, Randomized, Open-Label, Controlled Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Preterm Neonates 29 to 34 Weeks PMA: Dose Escalation and Study Extension

The primary objective of this study is to evaluate the safety and tolerability of aerosolized surfactant, specifically lucinactant for inhalation, administered in escalating inhaled doses to preterm neonates 29 to 34 weeks gestational age who are receiving nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome (RDS), compared to neonates receiving nCPAP alone.

Study Overview

• Status: Completed
• Conditions: Respiratory Distress Syndrome
• Intervention / Treatment: Drug: Lucinactant for Inhalation; Device: nCPAP alone

• Study Type: Interventional
• Enrollment (Actual): 80
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35249
      • University of Alabama at Birmingham
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Loma Linda, California, United States, 92354
      • Loma Linda University Medical Center
    • San Diego, California, United States, 92123
      • Sharp Mary Birch Hospital for Women and Newborns
  • Delaware
    • Newark, Delaware, United States, 19713
      • Christiana Care Health System
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children at IU Health
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Louisville
  • New Jersey
    • Morristown, New Jersey, United States, 07960
      • Mid Atlantic Neonatology Associates
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Oregon
    • Portland, Oregon, United States, 97225
      • Providence St. Vincent Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 7 months (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Informed consent from a legally authorized representative
• Gestational age 29 to 34 completed weeks (34 weeks 6 days) post menstrual age (PMA)
• Successful implementation of controlled nCPAP within 90 minutes after birth
• Spontaneous breathing
• Chest radiograph consistent with RDS
• Need for moderate levels of supplemental oxygen and nCPAP to maintain oxygen saturation of 88% to 95% for at least 30 minutes within the first 21 hours after birth

Exclusion Criteria:

• Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth
• Recurrent episodes of apnea occurring after the initial newborn resuscitation period (ie, 10 minutes after birth) requiring intermittent positive pressure breaths using inflating pressures above the set CPAP pressure administered manually or mechanically through any patient interface
• A 5 minute Apgar score < 5
• Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth
• Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH)
• Known or suspected chromosomal abnormality or syndrome
• Prolong rupture of membranes (PROM) > 2 weeks
• Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis
• Need for endotracheal intubation and mechanical ventilation
• Has been administered: another investigational agent or exposure to an investigational medical device, any other surfactant agent, steroid treatment after birth

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: SEQUENTIAL
• Masking: NONE

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Aerosolized lucinactant (25 mg/kg); 25 mg total phospholipids (TPL)/kg: Lucinactant for inhalation with nCPAP	Drug: Lucinactant for Inhalation; Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product); Other Names: Aerosurf
EXPERIMENTAL: Aerosolized lucinactant (50 mg/kg); 50 mg TPL/kg: Lucinactant for inhalation with nCPAP	Drug: Lucinactant for Inhalation; Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product); Other Names: Aerosurf
EXPERIMENTAL: Aerosolized lucinactant (75 mg/kg); 75 mg TPL/kg: Lucinactant for inhalation with nCPAP	Drug: Lucinactant for Inhalation; Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product); Other Names: Aerosurf
EXPERIMENTAL: Aerosolized lucinactant (100 mg/kg); 100 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met.	Drug: Lucinactant for Inhalation; Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product); Other Names: Aerosurf
EXPERIMENTAL: Aerosolized lucinactant (150 mg/kg); 150 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met.	Drug: Lucinactant for Inhalation; Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product); Other Names: Aerosurf
ACTIVE_COMPARATOR: nCPAP alone; nCPAP therapy alone	Device: nCPAP alone; nCPAP therapy; Other Names: nasal continuous positive airway pressure

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Peri-Dosing Events	Pre-specified adverse events that occured during treatment administration; does not include nCPAP alone	Within 48 Hours after Initiation of Study Treatment
All Cause Mortality		Within 36 Weeks PMA
Oxygen Saturation Levels	Oxygen saturation as determined by pulse oximetry	Within 3 Hours of Randomization
Serum Electrolytes		24 Hours Post Randomization

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PCO2	Arterial carbon dioxide	Within 3 Hours of Randomization

Collaborators and Investigators

• Sponsor: Windtree Therapeutics
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
• Investigators: Principal Investigator: Robert Segal, MD, FACP, Windtree Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start: February 1, 2014
• Primary Completion (ACTUAL): October 1, 2015
• Study Completion (ACTUAL): November 1, 2015

Study Registration Dates

• First Submitted: February 25, 2014
• First Submitted That Met QC Criteria: February 26, 2014
• First Posted (ESTIMATE): February 28, 2014

Study Record Updates

• Last Update Posted (ACTUAL): April 21, 2017
• Last Update Submitted That Met QC Criteria: March 10, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Infant, Newborn, Diseases; Infant, Premature, Diseases; Respiratory Distress Syndrome; Respiratory Distress Syndrome, Newborn
• Other Study ID Numbers: 03-CL-1201; 4R44HL107000-02 (NIH)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO