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

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ClinicalTrials.gov Identifier: NCT04264156
Recruitment Status : Suspended (Corporate business reasons)
First Posted : February 11, 2020
Last Update Posted : January 14, 2021
Sponsor:
Information provided by (Responsible Party):
Windtree Therapeutics

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.

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Study Type : Interventional  (Clinical Trial)
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.
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 : April 18, 2020
Estimated Primary Completion Date : January 31, 2021
Estimated Study Completion Date : February 28, 2021


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 Name: 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




Primary Outcome Measures :
  1. Number of Participants with Respiratory Failure or Death [ Time Frame: 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 :
  1. Number of Participants with Respiratory Failure due to RDS [ Time Frame: 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

  2. Time to respiratory failure due to RDS [ Time Frame: 72 hours and 28 days of life ]
    Time at which intubation for mechanical ventilation and/or surfactant administration occurred

  3. Number of participants with bronchopulmonary dysplasia [ Time Frame: 36 weeks PMA ]
    Number of participants with bronchopulmonary dysplasia

  4. Number of participants alive and without evidence of BPD [ Time Frame: 36 weeks PMA ]
  5. All-cause mortality [ Time Frame: 36 weeks PMA or 28 days of life (whichever is later) ]
    All-cause mortality

  6. Number of Participants with Common Complications of Prematurity [ Time Frame: 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.

  7. Length of time subjects received mechanical ventilation and/or supplemental oxygen [ Time Frame: Through 36 weeks PMA ]
  8. Changes in PCO2 gas exchange [ Time Frame: Through 7 days of life ]
    Changes in PCO2 (through 72 hours only)

  9. Changes in gas exchange [ Time Frame: Through 7 days of life ]
    Changes in FiO2 (as determined by pulse oximetry)

  10. Re-hospitalizations post discharge [ Time Frame: Through 12 months corrected age ]
    Number of participants with, duration of, and reason for re-hospitalizations following initial discharge



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 6 Hours   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04264156


Locations
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Poland
Szpital Specjalistyczny nr 2 w Bytomiu Oddzial Noworodkow Blok Va
Bytom, Poland, 41-902
Ginekologiczno-Polozniczy Szpital Kliniczny Uniwewersytetu Medycznego im. Karola Marcinkowskiego
Poznan, Poland, 60-535
Samodzielny Publiczny Spcjalistyczny Zaklad Opieki Zdrowotnej "Zdroje", Oddzial Noworodkow
Szczecin, Poland, 70-780
Sponsors and Collaborators
Windtree Therapeutics
Investigators
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Principal Investigator: Carlos Guardia, MD Windtree Therapeutics, Inc.
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Responsible Party: Windtree Therapeutics
ClinicalTrials.gov Identifier: NCT04264156    
Other Study ID Numbers: 03-CL-1702
First Posted: February 11, 2020    Key Record Dates
Last Update Posted: January 14, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by Windtree Therapeutics:
intubation
surfactant
continuous positive airway pressure
aerosol
respiratory distress syndrome
Additional relevant MeSH terms:
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Respiratory Distress Syndrome, Newborn
Respiratory Distress Syndrome, Adult
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders
Infant, Premature, Diseases
Infant, Newborn, Diseases