Clinical trial NCT02636868: The Safety and Efficacy of Lucinactant for Inhalation in Premature 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 in Preterm Neonates 26 to 32 Weeks Gestational Age With Respiratory Distress Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Respiratory Distress Syndrome
Lead sponsor: Windtree Therapeutics
Sponsor class: INDUSTRY
First posted: 2015-12-22
Enrollment: 221
Sex: ALL
Minimum age (years): 26
Maximum age (years): 32
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to evaluate the safety and efficacy of lucinactant for inhalation administered as an aerosolized dose in two doses to preterm neonates 26 - 32 weeks gestational age who are receiving nasal continuous positive airway pressure (nCPAP) for Respiratory Distress Syndrome (RDS) compared to neonates receiving nCPAP alone.

Detailed description: The purpose of this study is to investigate the safety and efficacy of lucinactant for inhalation in preterm neonates 26 to 32 completed weeks post-menstrual age (PMA). Efficacy and safety are based on clinical evaluations. The endpoints specified are similar to those in Protocols 03-CL-1201 and 03-CL-1401 to allow for potential comparison and pooling of results.

The objective of this study is to evaluate the safety and efficacy of lucinactant for inhalation in conjunction with nCPAP, compared to nCPAP alone, in preterm neonates with RDS, as assessed by the time to and incidence of respiratory failure and/or death due to RDS over the first 72 hours of life, the incidence of bronchopulmonary dysplasia (BPD) at 36 weeks PMA, and change in physiologic parameters (FiO2 and PCO2) over the first 72 hours of life.

Eligibility criteria: Inclusion Criteria:

1. Signed informed consent form (ICF) from legally authorized representative
2. 26 0/7 to 32 6/7 completed weeks gestation PMA
3. Successful implementation of non-invasive support or ventilation within 90 minutes after birth
4. Spontaneous breathing
5. Chest radiograph consistent with RDS
6. Within the first 20 hours after birth requires an nCPAP of 5 to 7 centimeters water (cmH2O) with a fraction of inspired oxygen (FiO2) of ≥ 0.25 (\>0.21 for neonates 26-28 weeks PMA) to 0.40 that is clinically indicated for at least 30 minutes to maintain oxygen by pulse oximetry (SpO2) of 90% to 95%. Transient (\<10 minutes) FiO2 excursions outside this range do not reset the 30-minute requirement.

Exclusion Criteria:

1. A heart rate that cannot be stabilized above 100 beats per minute (bpm) within 5 minutes of birth
2. Recurrent episodes of apnea requiring positive pressure ventilation (PPV) administered manually or mechanically through any patient interface
3. A 5 minute Apgar score \< 5
4. Major congenital malformation(s) or craniofacial abnormalities that preclude the use of nCPAP, diagnosed antenatally or immediately after birth
5. Clinically significant diseases or conditions other than RDS which could potentially interfere with cardiopulmonary function (e.g. congenital heart disease, hydrops fetalis or congenital infection)
6. A known or suspected chromosomal abnormality or syndrome
7. Premature rupture of membranes (PROM) \> 3 weeks
8. Hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis
9. A need for intubation and/or mechanical ventilation at any time before enrollment into the study
10. The administration (or plan for administration) of any the following:

    * Another investigational agent or investigational medical device
    * Any other surfactant agent
    * Systemic corticosteroids (other than antenatal steroids already received)
11. Presence of air leak (pneumothorax, pneumomediastinum, pneumopericardium, subcutaneous emphysema, or definite evidence of pulmonary interstitial emphysema (PIE)) on the baseline chest radiograph

Primary outcomes:
measure: Number of Participants With Respiratory Failure or Death Due to Respiratory Distress Syndrome (RDS)
timeFrame: 72 hours
Secondary outcomes:
measure: Incidence of Respiratory Failure or Death Due to RDS
timeFrame: 72 hours
measure: Time to nCPAP Failure
timeFrame: 72 hours
measure: Incidence of Respiratory Failure or Death Due to RDS With Poisson Distribution Modeling
timeFrame: 72 hours
measure: Incidence of Respiratory Failure or Death Due to RDS
timeFrame: 28 days
measure: Number of Participants With Bronchopulmonary Dysplasia (BPD)
timeFrame: 36 weeks post-menstrual age (PMA)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Little Rock
country: United States
facility: Univ. of Arkansas Medical Center
state: Arkansas
city: Loma Linda
country: United States
facility: Loma Linda University Medical Center
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: San Diego
country: United States
facility: Sharp Mary Birch Hospital for Women and Newborns
state: California
city: Newark
country: United States
facility: Christiana Care Health System
state: Delaware
city: Miami
country: United States
facility: University of Miami Holtz Children's Hospital
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Albany
country: United States
facility: Albany Medical Center
state: New York
city: New York
country: United States
facility: Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY)
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: Brody School of Medicine at ECU
state: North Carolina
city: Wilmington
country: United States
facility: New Hanover Regional Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Case Western Reserve University (Rainbow Babies Hosp.)
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Providence St. Vincent Medical Center
state: Oregon
city: Providence
country: United States
facility: Women and Infants Hospital
state: Rhode Island
city: Dallas
country: United States
facility: Baylor University Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Hospital
state: Texas
city: Fort Worth
country: United States
facility: Texas Health Harris Methodist Hospital
state: Texas
city: Calgary
country: Canada
facility: Foothills Medical Centre
state: Alberta
city: Edmonton
country: Canada
facility: Royal Alexandra Hospital
state: Alberta
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
state: Ontario
city: Montreal
country: Canada
facility: Montreal Children's Hospital
state: Quebec
city: Montreal
country: Canada
facility: Sainte Justine Hospital
state: Quebec
city: Santiago
country: Chile
facility: Hospital Dr Sotero Del Rio
state: Region-MetropolitanadeSantiago
city: Concepción
country: Chile
facility: Hospital Clínico Regional de Concepción Dr Guillermo Grant Benavente
city: Santiago
country: Chile
facility: Hospital San Jose
city: Santiago
country: Chile
facility: Clinica Alamena de Santiago
city: Santiago
country: Chile
facility: Hospital Santiago Oriente Dr Luis Tisné Brousse
city: Santiago
country: Chile
facility: Hospital Clinico de la Pontificia Universidad Catolica de Chile
city: Santiago
country: Chile
facility: Hospital San Juan de Dios
city: Medellin
country: Colombia
facility: Fundacion Hospitalaria San Vicente de Paul
state: Antioquia
city: Medellin
country: Colombia
facility: Hospital General de Medellin
state: Antioquia
city: Cali
country: Colombia
facility: Fundacion Valle Del Lili
state: Valle Del Cauca
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Debrecen
country: Hungary
facility: Csolnoky Ferenc Korhaz
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Kozpont
city: Miskolc
country: Hungary
facility: Borsod-Abauj-Zemplen Megyei Korhaz es Egyetemi Okato
city: Nyiregyhaza
country: Hungary
facility: Szabolcs-Szatmar-Bereg Megyei Korhazak es Egyetemi Oktatokorhaz
city: Cork
country: Ireland
facility: Cork University Hospital
city: Limerick
country: Ireland
facility: Mid-Western Regional Hospital Limerick
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center
city: Wroclaw
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
state: Dolnoslaskie
city: Bydgoszcz
country: Poland
facility: S.U. nr2im. Dr. Jana Biziela Oddzial Kliniczny N. W. Z. Intensywna Terapia Noworodka wraz z Wgjazdowy m Zespolem N
state: Kujawsko-pomorksie
city: Lodz
country: Poland
facility: Instytut Centrum Zdrowja Matki Polki Klinika Neonatologii
state: Lodzkie
city: Krakow
country: Poland
facility: SP ZOZ Szpital Uniwersytecki w Krakowie, Oddzial Neonatologii
state: Malopolskie
city: Warsaw
country: Poland
facility: Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii
state: Mazowieckie
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
state: Pomorskie
city: Bytom
country: Poland
facility: Szpital Spegialistycszny nr 2 w Bytomia Oddzial Noworodkow Blok V
state: Slaskie
city: Szczecin
country: Poland
facility: Samodzielny Publiczny Specjalistczny Zaklad Opieki Zdrowotnej Zdroje
state: West Pomerania
city: Poznan
country: Poland
facility: Ginekologiczno-Polozniczy Szpital Klinicznym UM im. Karola Marcinkowskiego w Poznan i u Katedra Neonatologii
state: Wielkopolskie
Study officials:
affiliation: Windtree Therapeutics
name: Steven Simonson, MD
role: STUDY_DIRECTOR
References: