Clinical trial NCT02528318: Trial To Assess The Safety And Tolerability Of Lucinactant For Inhalation In Preterm Neonates 26 to 28 Weeks PMA
Official title: A Multicenter, Randomized, Open-label, Controlled Trial To Assess The Safety And Tolerability Of Lucinactant For Inhalation In Preterm Neonates 26 to 28 Weeks PMA
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Respiratory Distress Syndrome
Lead sponsor: Windtree Therapeutics
Sponsor class: INDUSTRY
First posted: 2015-08-19
Enrollment: 48
Sex: ALL
Minimum age (years): 26
Maximum age (years): 28
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: This study is to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in up to four escalating doses to preterm neonates 26 to 28 weeks gestational age who are receiving nCPAP for RDS compared to neonates receiving nCPAP alone.

Detailed description: This study was a multicenter, randomized, controlled, open-label, dose-escalation study, conducted to evaluate the safety and tolerability of lucinactant for inhalation in conjunction with nasal continuous positive airway pressure (nCPAP) in comparison with nCPAP alone. The study was to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in 4 escalating doses.

For this study, lucinactant for inhalation refers to the active investigational agent, lyophilized lucinactant, in combination with the prototype investigational delivery device. Reconstituted lyophilized lucinactant was aerosolized by the investigational device and introduced into the nCPAP circuit. Those randomized to the control arm continued to receive nCPAP alone. Dose assignments were unblinded, as the primary objective of this study was safety and tolerability.

Preterm neonates with respiratory distress syndrome (RDS) between 26 and 28 completed weeks PMA who were within the first 20 hours after birth and who had successful implementation of controlled nCPAP within 90 minutes of birth were considered to be potential subjects. Before study enrollment, legal guardians were provided a written informed consent form (ICF) for each potential subject.

Eligibility criteria: Inclusion Criteria:

1. Informed consent from a legally authorized representative.
2. Gestational age 26 to 28 completed weeks post menstrual age (PMA).
3. Successful implementation of controlled nCPAP within 90 minutes after birth.
4. Spontaneous breathing.
5. Chest radiograph consistent with RDS.
6. Within the first 20 hours after birth, have an nCPAP of 5 to 6 cm H2O to maintain oxygen saturation measured by pulse oximetry (SpO2) of 88% to 95% with a fraction of inspired oxygen (FiO2) of 0.25 to 0.50 that is clinically indicated for at least 30 minutes. Transient (\<10 minutes) FiO2 excursions below 0.25 or above 0.50 did not reset the 30 minute requirement.

Exclusion Criteria:

1. Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth.
2. Recurrent episodes of apnea occurring after the initial newborn resuscitation period (ie, 10 minutes after birth) requiring intermittent positive pressure breaths using inflating pressures above the set CPAP pressure administered manually or mechanically through any patient interface.
3. A 5 minute Apgar score \< 5.
4. Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth.
5. Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH).
6. Known or suspected chromosomal abnormality or syndrome.
7. Premature rupture of membranes (PROM) \> 2 weeks.
8. Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.
9. Need for endotracheal intubation and mechanical ventilation.
10. Has been administered: another investigational agent or investigational medical device, any other surfactant agent, steroid treatment after birth.

Primary outcomes:
measure: Number of Participants With Peri-Dosing Adverse Events - Initial Dose
timeFrame: Randomization to 24 Hours Post Randomization
measure: Number of Participants With Air Leak
timeFrame: 7 days
Secondary outcomes:
measure: Number of Participants With Worsening of Respiratory Status Criteria
timeFrame: Randomization to 72 Hours Post Randomization
measure: Bronchopulmonary Dysplasia
timeFrame: Randomization to 36 weeks PMA
measure: Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure
timeFrame: Randomization to 72 Hours Post Randomization
measure: Death
timeFrame: Randomization to 36 weeks PMA
measure: FiO2
timeFrame: Randomization to 72 hours post randomization
measure: Number of Participants With Complications of Prematurity
timeFrame: Randomization to 36 weeks PMA
Other outcomes:
measure: nCPAP Failure Without Treatment Interruptions
timeFrame: Randomization to 72 Hours Post Randomization
Locations:
city: Loma Linda
country: United States
facility: Loma Linda University Medical Center
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
state: Florida
city: Louisville
country: United States
facility: University of Louisville
state: Kentucky
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: New York
country: United States
facility: Columbia University College of Physicians and Surgeons, New York Presbyterian Hospital - Morgan Stanley Children's Hospital
state: New York
city: Wilmington
country: United States
facility: New Hanover Regional Medical Center
state: North Carolina
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: Calgary
country: Canada
facility: Foothills Medical Centre
state: Alberta
city: Edmonton
country: Canada
facility: Royal Alexandria 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: Santiago
country: Chile
facility: Hospital Dr Sotero Del Rio
city: Santiago
country: Chile
facility: Hospital San Juan de Dios
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: Warsaw
country: Poland
facility: Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii
state: Mazowieckie
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: Steve Simonson, MD
role: STUDY_DIRECTOR
References: