The Safety and Efficacy of Lucinactant for Inhalation in Premature Neonates 26 to 32 Weeks Gestational Age

NCT02636868 | Completed Phase 2 Results DMC

• 1 other identifier: 03-CL-1202
• Study Type: interventional
• Target: 221
• Locations: 8 countries
• Sites: 54

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.

Conditions

Respiratory Distress Syndrome

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Respiratory Failure or Death Due to Respiratory Distress Syndrome (RDS)

  Number of participants who had respiratory failure due to RDS or death due to RDS; known as nasal continuous positive airway pressure (nCPAP) failure

  72 hours

Secondary Outcomes (5)

• Incidence of Respiratory Failure or Death Due to RDS

  72 hours

• Time to nCPAP Failure

  72 hours

• Incidence of Respiratory Failure or Death Due to RDS With Poisson Distribution Modeling

  72 hours

• Incidence of Respiratory Failure or Death Due to RDS

  28 days

• Number of Participants With Bronchopulmonary Dysplasia (BPD)

  36 weeks post-menstrual age (PMA)

Study Arms (3)

Aerosolized lucinactant (low dose)

EXPERIMENTAL

Lucinactant for inhalation with nCPAP; up to 2 repeat doses will be allowed if repeat dosing criteria are met.

Drug: Lucinactant delivered via investigational delivery device; Drug: nCPAP

Aerosolized lucinactant (high dose)

EXPERIMENTAL

Lucinactant for inhalation with nCPAP; up to 2 repeat doses will be allowed if repeat dosing criteria are met.

Drug: Lucinactant delivered via investigational delivery device; Drug: nCPAP

nasal CPAP

ACTIVE COMPARATOR

nCPAP alone

Drug: nCPAP

Interventions

Lucinactant delivered via investigational delivery device DRUG

Lucinactant for inhalation refers to the active investigational agent lucinactant in combination with the investigational delivery device (drug-device combination product)

Also known as: AEROSURF

Aerosolized lucinactant (high dose); Aerosolized lucinactant (low dose)

nCPAP DRUG

Nasal CPAP

Aerosolized lucinactant (high dose); Aerosolized lucinactant (low dose); nasal CPAP

Eligibility Criteria

• Age: 26 Weeks - 32 Weeks
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Signed informed consent form (ICF) from legally authorized representative
• /7 to 32 6/7 completed weeks gestation PMA
• Successful implementation of non-invasive support or ventilation within 90 minutes after birth
• Spontaneous breathing
• Chest radiograph consistent with RDS
• 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.

You may not qualify if:

• A heart rate that cannot be stabilized above 100 beats per minute (bpm) within 5 minutes of birth
• Recurrent episodes of apnea requiring positive pressure ventilation (PPV) administered manually or mechanically through any patient interface
• A 5 minute Apgar score \< 5
• Major congenital malformation(s) or craniofacial abnormalities that preclude the use of nCPAP, diagnosed antenatally or immediately after birth
• 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)
• A known or suspected chromosomal abnormality or syndrome
• Premature rupture of membranes (PROM) \> 3 weeks
• Hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis
• A need for intubation and/or mechanical ventilation at any time before enrollment into the study
• 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)
• Presence of air leak (pneumothorax, pneumomediastinum, pneumopericardium, subcutaneous emphysema, or definite evidence of pulmonary interstitial emphysema (PIE)) on the baseline chest radiograph

Sponsors & Collaborators

• Windtree Therapeutics: lead

Study Sites (54)

University of Alabama at Birmingham

Birmingham, Alabama, 35249, United States

Location

Univ. of Arkansas Medical Center

Little Rock, Arkansas, 72202, United States

Location

Loma Linda University Medical Center

Loma Linda, California, 92354, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Sharp Mary Birch Hospital for Women and Newborns

San Diego, California, 92123, United States

Location

Christiana Care Health System

Newark, Delaware, 19713, United States

Location

University of Miami Holtz Children's Hospital

Miami, Florida, 33136, United States

Location

University of Michigan Health System

Ann Arbor, Michigan, 48109, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68105, United States

Location

Albany Medical Center

Albany, New York, 12208, United States

Location

Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY)

New York, New York, 10032, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Brody School of Medicine at ECU

Greenville, North Carolina, 27834, United States

Location

New Hanover Regional Medical Center

Wilmington, North Carolina, 28401, United States

Location

Case Western Reserve University (Rainbow Babies Hosp.)

Cleveland, Ohio, 44106, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Providence St. Vincent Medical Center

Portland, Oregon, 97225, United States

Location

Women and Infants Hospital

Providence, Rhode Island, 02905, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Cook Children's Hospital

Fort Worth, Texas, 76104, United States

Location

Texas Health Harris Methodist Hospital

Fort Worth, Texas, 76104, United States

Location

Foothills Medical Centre

Calgary, Alberta, T2N 2T9, Canada

Location

Royal Alexandra Hospital

Edmonton, Alberta, T5H 3V9, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Montreal Children's Hospital

Montreal, Quebec, H3H 1P3, Canada

Location

Sainte Justine Hospital

Montreal, Quebec, H3T 1C5, Canada

Location

Hospital Dr Sotero Del Rio

Santiago, Region-MetropolitanadeSantiago, 8207257, Chile

Location

Hospital Clínico Regional de Concepción Dr Guillermo Grant Benavente

Concepción, 4070038, Chile

Location

Hospital San Jose

Santiago, 3330, Chile

Location

Clinica Alamena de Santiago

Santiago, 7650568, Chile

Location

Hospital Santiago Oriente Dr Luis Tisné Brousse

Santiago, 7980378, Chile

Location

Hospital Clinico de la Pontificia Universidad Catolica de Chile

Santiago, 8330024, Chile

Location

Hospital San Juan de Dios

Santiago, 8350488, Chile

Location

Fundacion Hospitalaria San Vicente de Paul

Medellín, Antioquia, 050010, Colombia

Location

Hospital General de Medellin

Medellín, Antioquia, 050015, Colombia

Location

Fundacion Valle Del Lili

Cali, Valle del Cauca Department, 760032, Colombia

Location

Semmelweis Egyetem

Budapest, 1082, Hungary

Location

Csolnoky Ferenc Korhaz

Debrecen, 4032, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, H-4012, Hungary

Location

Borsod-Abauj-Zemplen Megyei Korhaz es Egyetemi Okato

Miskolc, 3526, Hungary

Location

Szabolcs-Szatmar-Bereg Megyei Korhazak es Egyetemi Oktatokorhaz

Nyíregyháza, H-4400, Hungary

Location

Cork University Hospital

Cork, T12 YN60, Ireland

Location

Mid-Western Regional Hospital Limerick

Limerick, Ireland

Location

Erasmus Medical Center

Rotterdam, 3000 CB, Netherlands

Location

Ginekologiczno-Polozniczy Szpital Klinicznym UM im. Karola Marcinkowskiego w Poznan i u Katedra Neonatologii

Poznan, Greater Poland Voivodeship, 60-535, Poland

Location

S.U. nr2im. Dr. Jana Biziela Oddzial Kliniczny N. W. Z. Intensywna Terapia Noworodka wraz z Wgjazdowy m Zespolem N

Bydgoszcz, Kujawsko-pomorksie, 85-168, Poland

Location

SP ZOZ Szpital Uniwersytecki w Krakowie, Oddzial Neonatologii

Krakow, Lesser Poland Voivodeship, 31-501, Poland

Location

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu

Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

Location

Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii

Warsaw, Masovian Voivodeship, 00-315, Poland

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, Pomeranian Voivodeship, 80-402, Poland

Location

Szpital Spegialistycszny nr 2 w Bytomia Oddzial Noworodkow Blok V

Bytom, Silesian Voivodeship, 41-902, Poland

Location

Samodzielny Publiczny Specjalistczny Zaklad Opieki Zdrowotnej Zdroje

Szczecin, West Pomeranian Voivodeship, 70-780, Poland

Location

Instytut Centrum Zdrowja Matki Polki Klinika Neonatologii

Lodz, Łódź Voivodeship, 93-338, Poland

Location

MeSH Terms

Conditions

Respiratory Distress Syndrome

Interventions

lucinactant

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Limitations and Caveats

Clogging of a in-line filter led to a higher number of treatment interruptions than expected. This primarily affected one batch of supplies that were, by chance, predominantly used in European sites.

Results Point of Contact

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

psimmons@windtreetx.com

215-488-9477

Study Officials

• Steven Simonson, MD

  Windtree Therapeutics

  STUDY DIRECTOR

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: CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2015
• First Posted: December 22, 2015
• Study Start: December 1, 2015
• Primary Completion: July 1, 2017
• Study Completion: August 6, 2019
• Last Updated: April 23, 2021
• Results First Posted: April 23, 2021

Record last verified: 2021-03

Locations

PL (9); CL (7); HU (5); NL (1); CO (3); IE (2); US (22); CA (5)