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

Primary Purpose

Respiratory Distress Syndrome, Newborn

Status

Terminated

Phase

Phase 2

Locations

Poland

Study Type

Interventional

Intervention

Lucinactant for Inhalation

nCPAP Only

About this trial

This is an interventional treatment trial for Respiratory Distress Syndrome, Newborn focused on measuring intubation, surfactant, continuous positive airway pressure, aerosol, respiratory distress syndrome

Eligibility Criteria

30 Minutes - 6 Hours (Child)All SexesDoes not accept 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.

Sites / Locations

Szpital Specjalistyczny nr 2 w Bytomiu Oddzial Noworodkow Blok Va
Ginekologiczno-Polozniczy Szpital Kliniczny Uniwewersytetu Medycznego im. Karola Marcinkowskiego
Samodzielny Publiczny Spcjalistyczny Zaklad Opieki Zdrowotnej "Zdroje", Oddzial Noworodkow

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Sham Comparator

Arm Label

Lucinactant (160 mg/kg) + nCPAP

nCPAP Only

Arm Description

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

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

Outcomes

Primary Outcome Measures

Number of Participants With Respiratory Failure or Death

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 With BPD

Number of participants with bronchopulmonary dysplasia (BPD)

Mortality

All-cause mortality

Number of Participants With Common Complications of Prematurity

Number of participants with complications including intraventricular hemorrhage, periventricular leukomalacia, pulmonary hemorrhage, apnea, necrotizing enterocolitis, patent ductus arteriosus, acquired sepsis, and retinopathy of prematurity.

Full Information

NCT ID

NCT04264156

First Posted

February 7, 2020

Last Updated

April 27, 2023

Sponsor

Windtree Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT04264156

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Terminated

Why Stopped

Corporate business reasons

Study Start Date

April 18, 2020 (Actual)

Primary Completion Date

January 31, 2021 (Actual)

Study Completion Date

March 28, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Windtree Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Yes

Device Product Not Approved or Cleared by U.S. FDA

Yes

Data Monitoring Committee

Yes

5. 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).

Detailed Description

An unmet medical need exists for a means to deliver surfactant replacement therapy (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 mechanical ventilation (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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Respiratory Distress Syndrome, Newborn

Keywords

intubation, surfactant, continuous positive airway pressure, aerosol, respiratory distress syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

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

Masking

ParticipantCare ProviderInvestigator

Masking Description

The first 2 subjects at each site for each cohort will be dosed with open-label active treatment for training purposes. 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, parents, or other applicable study staff.

Allocation

Randomized

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Lucinactant (160 mg/kg) + nCPAP

Arm Type

Experimental

Arm Description

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

Arm Title

nCPAP Only

Arm Type

Sham Comparator

Arm Description

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

Intervention Type

Combination Product

Intervention Name(s)

Lucinactant for Inhalation

Other Intervention Name(s)

AEROSURF

Intervention Description

A drug-device combination product that delivers aerosolized SRT to preterm neonates with RDS who are being supported with nasal continuous positive airway pressure (nCPAP).

Intervention Type

Other

Intervention Name(s)

nCPAP Only

Intervention Description

Nasal continuous positive airway pressure (nCPAP) alone

Primary Outcome Measure Information:

Title

Number of Participants With Respiratory Failure or Death

Description

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

Time Frame

28 days of life

Secondary Outcome Measure Information:

Title

Number With BPD

Description

Number of participants with bronchopulmonary dysplasia (BPD)

Time Frame

36 weeks post-menstrual age (PMA)

Title

Mortality

Description

All-cause mortality

Time Frame

36 weeks PMA or 28 days of life (whichever is later)

Title

Number of Participants With Common Complications of Prematurity

Description

Time Frame

36 weeks PMA

10. Eligibility

Sex

All

Minimum Age & Unit of Time

30 Minutes

Maximum Age & Unit of Time

6 Hours

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Carlos Guardia, MD

Organizational Affiliation

Windtree Therapeutics, Inc.

Official's Role

Principal Investigator

Facility Information:

Facility Name

Szpital Specjalistyczny nr 2 w Bytomiu Oddzial Noworodkow Blok Va

City

Bytom

ZIP/Postal Code

41-902

Country

Poland

Facility Name

Ginekologiczno-Polozniczy Szpital Kliniczny Uniwewersytetu Medycznego im. Karola Marcinkowskiego

City

Poznan

ZIP/Postal Code

60-535

Country

Poland

Facility Name

Samodzielny Publiczny Spcjalistyczny Zaklad Opieki Zdrowotnej "Zdroje", Oddzial Noworodkow

City

Szczecin

ZIP/Postal Code

70-780

Country

Poland

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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