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

NCT04264156 | Terminated Phase 2 Results DMC FDA Drug FDA Device

• 1 other identifier: 03-CL-1702
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

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).

Conditions

Respiratory Distress Syndrome, Newborn

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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

  28 days of life

Secondary Outcomes (3)

• Number With BPD

  36 weeks post-menstrual age (PMA)

• Mortality

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

• Number of Participants With Common Complications of Prematurity

  36 weeks PMA

Study Arms (2)

Lucinactant (160 mg/kg) + nCPAP

EXPERIMENTAL

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

nCPAP Only

SHAM COMPARATOR

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

Other: nCPAP Only

Interventions

Lucinactant for Inhalation COMBINATION_PRODUCT

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).

Also known as: AEROSURF

Lucinactant (160 mg/kg) + nCPAP

nCPAP Only OTHER

Nasal continuous positive airway pressure (nCPAP) alone

nCPAP Only

Eligibility Criteria

• Age: 30 Minutes - 6 Hours
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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%.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Windtree Therapeutics: lead

Study Sites (3)

Szpital Specjalistyczny nr 2 w Bytomiu Oddzial Noworodkow Blok Va

Bytom, 41-902, Poland

Location

Ginekologiczno-Polozniczy Szpital Kliniczny Uniwewersytetu Medycznego im. Karola Marcinkowskiego

Poznan, 60-535, Poland

Location

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

Szczecin, 70-780, Poland

Location

MeSH Terms

Conditions

Respiratory Distress Syndrome, Newborn; Respiratory Distress Syndrome

Interventions

lucinactant; Inhalation

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Respiratory Mechanics; Respiration; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena

Limitations and Caveats

Study was terminated early leading to a very small number of subjects analyzed.

Results Point of Contact

• Title: Executive Director of Biostatistics & Data Management
• Organization: Windtree Therapeutics, Inc.

psimmons@windtreetx.com

2154889300

Study Officials

• Carlos Guardia, MD

  Windtree Therapeutics, Inc.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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).

Study Record Dates

• First Submitted: February 7, 2020
• First Posted: February 11, 2020
• Study Start: April 18, 2020
• Primary Completion: January 31, 2021
• Study Completion: March 28, 2021
• Last Updated: May 24, 2023
• Results First Posted: May 24, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

PL (3)