A Safety and Efficacy Study of Lucinactant for Inhalation in Preterm Neonates 26 to 32 Weeks GA

Sponsor

Windtree Therapeutics (Industry)

Overall Status

Suspended

CT.gov ID

NCT04264156

Collaborator

(none)

Enrollment

Locations

Arms

10.4

Anticipated Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is to evaluate the safety and efficacy of lucinactant for inhalation in conjunction with nCPAP, in comparison to nCPAP alone, in preterm neonates with RDS, as assessed by the incidence of and time to respiratory failure and/or death due to RDS in the first 72 hours and 28 days of life. Half of the subjects will receive lucinactant for inhalation and half will receive standard of care (nCPAP alone).

Condition or Disease	Intervention/Treatment	Phase
Respiratory Distress Syndrome, Newborn	Combination Product: Lucinactant for Inhalation Other: nCPAP Only	Phase 2

Detailed Description

An unmet medical need exists for a means to deliver SRT to preterm neonates with RDS supported with nCPAP early in the course of the disease. This strategy has the potential to improve RDS prior to the development of respiratory failure, thereby avoiding the need for endotracheal intubation and MV, or reduce the duration of MV, and the resultant potential for morbidity and complications. The ability to administer SRT via aerosol has the potential to address this unmet need.

Lucinactant for inhalation (AEROSURF) is an investigational drug-device combination product, designed to deliver aerosolized SRT to preterm neonates with RDS who are being supported with nCPAP. The drug component of lucinactant for inhalation is lyophilized lucinactant, a lyophilized form of SURFAXIN® (lucinactant) Intratracheal Suspension. The device component, the AEROSURF Delivery System (ADS), the next-generation device following use of the prototype device in earlier trials, uses novel technology to aerosolize lucinactant for inhalation.

This study evaluates the safety and efficacy of lucinactant for inhalation in conjunction with nCPAP, in comparison to nCPAP alone, in preterm neonates with RDS, as assessed by pre-specified outcome measures. In addition, this study will evaluate the device and the ability to administer up to 3 repeat doses.

Study Design

Study Type:

Interventional

Actual Enrollment :

12 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

This study is a multinational, multicenter, double-blind (masked), parallel group, randomized, controlled study, in preterm neonates 26 to 32 completed weeks PMA.This study is a multinational, multicenter, double-blind (masked), parallel group, randomized, controlled study, in preterm neonates 26 to 32 completed weeks PMA.

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

The first 2 subjects at each site will be dosed with open-label active treatment. Following the first 2 subjects, preparation and delivery of treatment will be blinded from the study staff. Treatment will be delivered behind a partition and no information about treatment will be given to investigator, outcomes assessor, parents, or other applicable study staff.

Primary Purpose:

Treatment

Official Title:

A Multinational, Multicenter, Masked, Randomized, Controlled Study to Assess the Safety and Efficacy of Lucinactant for Inhalation Versus nCPAP Alone in Preterm Neonates 26 to 32 Weeks Gestational Age With RDS

Actual Study Start Date :

Apr 18, 2020

Anticipated Primary Completion Date :

Jan 31, 2021

Anticipated Study Completion Date :

Feb 28, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 160 mg/kg Lucinactant for inhalation, 160 mg total phospholipids (TPL)/kg Delivered as an aerosol once, with up to 3 repeats of 80 mg/kg allowed within 36 hours of birth	Combination Product: Lucinactant for Inhalation A drug-device combination product that delivers aerosolized SRT to preterm neonates with RDS who are being supported with nCPAP. Other Names: AEROSURF
Sham Comparator: nCPAP Only nCPAP Only as sham comparator. Bubble nCPAP is standard of care. Treatment time behind barrier to match active treatment delivery time	Other: nCPAP Only Nasal CPAP Alone

Arm

Intervention/Treatment

Experimental: 160 mg/kg

Lucinactant for inhalation, 160 mg total phospholipids (TPL)/kg Delivered as an aerosol once, with up to 3 repeats of 80 mg/kg allowed within 36 hours of birth

Combination Product: Lucinactant for Inhalation

A drug-device combination product that delivers aerosolized SRT to preterm neonates with RDS who are being supported with nCPAP.

Other Names:

AEROSURF

Sham Comparator: nCPAP Only

nCPAP Only as sham comparator. Bubble nCPAP is standard of care. Treatment time behind barrier to match active treatment delivery time

Other: nCPAP Only

Nasal CPAP Alone

Outcome Measures

Primary Outcome Measures

Number of Participants with Respiratory Failure or Death [28 days of life]
Number of participants with respiratory failure due to RDS or death. Respiratory failure due to RDS is defined as intubation for mechanical ventilation and/or surfactant administration

Secondary Outcome Measures

Number of Participants with Respiratory Failure due to RDS [72 hours and 28 days of life]
Number of participants with respiratory failure due to RDS. Respiratory failure due to RDS is defined as intubation for mechanical ventilation and/or surfactant administration
Time to respiratory failure due to RDS [72 hours and 28 days of life]
Time at which intubation for mechanical ventilation and/or surfactant administration occurred
Number of participants with bronchopulmonary dysplasia [36 weeks PMA]
Number of participants with bronchopulmonary dysplasia
Number of participants alive and without evidence of BPD [36 weeks PMA]
All-cause mortality [36 weeks PMA or 28 days of life (whichever is later)]
All-cause mortality
Number of Participants with Common Complications of Prematurity [36 weeks PMA]
Number of participants with complications including intraventricular hemorrhage, periventricular leukomalacia, pulmonary hemorrhage, apnea, necrotizing enterocolitis, patent ductus arteriosus, acquired sepsis, and retinopathy of prematurity.
Length of time subjects received mechanical ventilation and/or supplemental oxygen [Through 36 weeks PMA]
Changes in PCO2 gas exchange [Through 7 days of life]
Changes in PCO2 (through 72 hours only)
Changes in gas exchange [Through 7 days of life]
Changes in FiO2 (as determined by pulse oximetry)
Re-hospitalizations post discharge [Through 12 months corrected age]
Number of participants with, duration of, and reason for re-hospitalizations following initial discharge

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 6 Hours

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Signed ICF from legally authorized representative.
Gestational age: 26 to 32+6 weeks PMA.
Successful implementation of non-invasive support or ventilation within 30 minutes after birth.
Spontaneous breathing.
Investigator determination of RDS. A chest x-ray should be obtained before treatment to confirm the diagnosis.
Within the first 6 hours after birth, requires an nCPAP of 5 to 7 cm H2O that is clinically indicated for at least 15 minutes with an FiO2 > 0.25 to ≤ 0.35 to maintain SpO2 of 90% to 95%.

Exclusion Criteria:

A heart rate that cannot be stabilized above 100 bpm within 5 minutes of birth.
Recurrent episodes of apnea requiring positive pressure ventilation.
A 5 minute Apgar score < 5.
Major congenital malformation(s) or craniofacial abnormalities that preclude the use of nCPAP.
Clinically significant diseases or conditions other than RDS which could potentially interfere with cardiopulmonary function.
A known or suspected chromosomal abnormality or syndrome.
Premature rupture of membranes > 3 weeks.
Hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.
A need for intubation and/or invasive mechanical ventilation at any time before enrollment into the study.
The administration (or plan for administration) of another investigational agent or investigational medical device, any other surfactant agent, or systemic corticosteroids.
Presence of air leak on the baseline chest radiograph or diagnosed via ultrasound or illumination.

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Szpital Specjalistyczny nr 2 w Bytomiu Oddzial Noworodkow Blok Va	Bytom	Poland	41-902
2	Ginekologiczno-Polozniczy Szpital Kliniczny Uniwewersytetu Medycznego im. Karola Marcinkowskiego	Poznan	Poland	60-535
3	Samodzielny Publiczny Spcjalistyczny Zaklad Opieki Zdrowotnej "Zdroje", Oddzial Noworodkow	Szczecin	Poland	70-780