Respiratory Distress SyndromePhase 2NCT02636868
The Safety and Efficacy of Lucinactant for Inhalation in Premature Neonates 26 to 32 Weeks Gestational Age
A Phase 2 study testing Lucinactant delivered via investigational delivery device and nCPAP in people with Respiratory Distress Syndrome, run by Windtree Therapeutics at 54 sites in 8 countries.
Trial 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)… Read more about this study
- Who is it for?
- Ages 26 to 32 weeks
- Conditions the study is for
- Respiratory Distress Syndrome
- What is being tested?
- Lucinactant delivered via investigational delivery device and nCPAP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 221 participants
- Where
- 54 sitesin 8 countries
- Study dates
- Dec 2015to 6 Aug 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 26 to 32 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Signed informed consent form (ICF) from legally authorized representative
- 26 0/7 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.
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
54 sites in 8 countries.
54 sites in 8 countries
United States22 sites
Albany Medical Center
Albany, New York, 12208
University of Michigan Health System
Ann Arbor, Michigan, 48109
University of Alabama at Birmingham
Birmingham, Alabama, 35249
Case Western Reserve University (Rainbow Babies Hosp.)
Cleveland, Ohio, 44106
Ohio State University
Columbus, Ohio, 43210
Baylor University Medical Center
Dallas, Texas, 75246
Duke University Medical Center
Durham, North Carolina, 27710
Cook Children's Hospital
Fort Worth, Texas, 76104
Texas Health Harris Methodist Hospital
Fort Worth, Texas, 76104
Brody School of Medicine at ECU
Greenville, North Carolina, 27834
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Univ. of Arkansas Medical Center
Little Rock, Arkansas, 72202
Loma Linda University Medical Center
Loma Linda, California, 92354
University of Miami Holtz Children's Hospital
Miami, Florida, 33136
Morgan Stanley Childrens Hospital of New York Presbyterian (CHONY)
New York, New York, 10032
Christiana Care Health System
Newark, Delaware, 19713
University of Nebraska Medical Center
Omaha, Nebraska, 68105
Children's Hospital of Orange County
Orange, California, 92868
Providence St. Vincent Medical Center
Portland, Oregon, 97225
Women and Infants Hospital
Providence, Rhode Island, 02905
Sharp Mary Birch Hospital for Women and Newborns
San Diego, California, 92123
New Hanover Regional Medical Center
Wilmington, North Carolina, 28401
Poland9 sites
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
Szpital Spegialistycszny nr 2 w Bytomia Oddzial Noworodkow Blok V
Bytom, Silesian Voivodeship, 41-902
Uniwersyteckie Centrum Kliniczne
Gdansk, Pomeranian Voivodeship, 80-402
SP ZOZ Szpital Uniwersytecki w Krakowie, Oddzial Neonatologii
Krakow, Lesser Poland Voivodeship, 31-501
Instytut Centrum Zdrowja Matki Polki Klinika Neonatologii
Lodz, Łódź Voivodeship, 93-338
Ginekologiczno-Polozniczy Szpital Klinicznym UM im. Karola Marcinkowskiego w Poznan i u Katedra Neonatologii
Poznan, Greater Poland Voivodeship, 60-535
Samodzielny Publiczny Specjalistczny Zaklad Opieki Zdrowotnej Zdroje
Szczecin, West Pomeranian Voivodeship, 70-780
Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii
Warsaw, Masovian Voivodeship, 00-315
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
Wroclaw, Lower Silesian Voivodeship, 50-556
Chile7 sites
Hospital Clínico Regional de Concepción Dr Guillermo Grant Benavente
Concepción, 4070038
Hospital Dr Sotero Del Rio
Santiago, Region-MetropolitanadeSantiago, 8207257
Hospital San Jose
Santiago, 3330
Clinica Alamena de Santiago
Santiago, 7650568
Hospital Santiago Oriente Dr Luis Tisné Brousse
Santiago, 7980378
Hospital Clinico de la Pontificia Universidad Catolica de Chile
Santiago, 8330024
Hospital San Juan de Dios
Santiago, 8350488
Canada5 sites
Foothills Medical Centre
Calgary, Alberta, T2N 2T9
Royal Alexandra Hospital
Edmonton, Alberta, T5H 3V9
Montreal Children's Hospital
Montreal, Quebec, H3H 1P3
Sainte Justine Hospital
Montreal, Quebec, H3T 1C5
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Hungary5 sites
Semmelweis Egyetem
Budapest, 1082
Csolnoky Ferenc Korhaz
Debrecen, 4032
Debreceni Egyetem Klinikai Kozpont
Debrecen, H-4012
Borsod-Abauj-Zemplen Megyei Korhaz es Egyetemi Okato
Miskolc, 3526
Szabolcs-Szatmar-Bereg Megyei Korhazak es Egyetemi Oktatokorhaz
Nyíregyháza, H-4400
Colombia3 sites
Fundacion Valle Del Lili
Cali, Valle del Cauca Department, 760032
Fundacion Hospitalaria San Vicente de Paul
Medellín, Antioquia, 050010
Hospital General de Medellin
Medellín, Antioquia, 050015
Ireland2 sites
Cork University Hospital
Cork, T12 YN60
Mid-Western Regional Hospital Limerick
Limerick
About this study
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
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.
Read the 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.
- Conditions
- Respiratory Distress Syndrome
- Being tested
- Lucinactant delivered via investigational delivery device, nCPAP
- Medical subject headings
- Respiratory Distress Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lucinactant delivered via investigational delivery deviceDrug
Also called AEROSURF
Lucinactant for inhalation refers to the active investigational agent lucinactant in combination with the investigational delivery device (drug-device combination product)
nCPAPDrug
Nasal CPAP
| Study group | Role | What they receive |
|---|---|---|
| Aerosolized lucinactant (low dose) | Experimental | Lucinactant for inhalation with nCPAP; up to 2 repeat doses will be allowed if repeat dosing criteria are met. Receives: 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. Receives: Drug: Lucinactant delivered via investigational delivery device; Drug: nCPAP |
| nasal CPAP | Active comparator | nCPAP alone Receives: Drug: nCPAP |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 72 hours
Secondary outcomes (5)
Incidence of Respiratory Failure or Death Due to RDS
Incidence of Respiratory Failure or Death Due to RDS by Intubation or Failure Criteria
Measured: 72 hours
Time to nCPAP Failure
Time from birth to nCPAP Failure
Measured: 72 hours
Incidence of Respiratory Failure or Death Due to RDS With Poisson Distribution Modeling
The measure tests the differences between treatments on respiratory failure or death due to RDS using Poisson distribution modeling, which accounts for the time over which the event could have occurred.
Measured: 72 hours
Incidence of Respiratory Failure or Death Due to RDS
Incidence of Respiratory Failure or Death due to RDS by Intubation or Failure Criteria
Measured: 28 days
Number of Participants With Bronchopulmonary Dysplasia (BPD)
Summarizes the number of participants with BPD or alive without BPD
Measured: 36 weeks post-menstrual age (PMA)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2015 (estimated)
- Study startDec 2015
- Primary completionJul 2017
- Study completion6 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Respiratory Distress SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Care provider, Investigator, Outcomes assessor
- Enrolment
- 221 participants (actual)
Sponsor and investigators
- Lead sponsor
- Windtree Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Steven Simonson, MD, Study director, Windtree Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02636868
- Sponsor's protocol ID
- 03-CL-1202
- Status last verified
- Mar 2021
- First submitted
- 19 Nov 2015
- Last updated
- 23 Apr 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02636868
- Acronym / sponsor ID
- 03-CL-1202
- Conditions
- Respiratory Distress Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 221 participants (actual)
- Lead sponsor
- Windtree TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2015 (actual)
- Primary completion
- Jul 2017 (actual)
- Study completion
- 6 Aug 2019 (actual)
- First posted
- 22 Dec 2015 (estimated)
- Results
- Posted 23 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Aerosolized lucinactant (low dose) | Experimental | Drug: Lucinactant delivered via investigational delivery device, Drug: nCPAPLucinactant for inhalation with nCPAP; up to 2 repeat doses will be allowed if repeat dosing criteria are met. |
| Aerosolized lucinactant (high dose) | Experimental | Drug: Lucinactant delivered via investigational delivery device, Drug: nCPAPLucinactant for inhalation with nCPAP; up to 2 repeat doses will be allowed if repeat dosing criteria are met. |
| nasal CPAP | Active comparator | Drug: nCPAPnCPAP alone |
Who may qualify
- Ages
- 26 to 32 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Signed informed consent form (ICF) from legally authorized representative
- 26 0/7 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
Key exclusion criteria (14 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lucinactant delivered via investigational delivery deviceAlso: AEROSURFLucinactant for inhalation refers to the active investigational agent lucinactant in combination with the investigational delivery device (drug-device combination product) | Drug | Aerosolized lucinactant (high dose), Aerosolized lucinactant (low dose) |
| nCPAPNasal CPAP | Drug | Aerosolized lucinactant (high dose), Aerosolized lucinactant (low dose), nasal CPAP |
Primary outcome
Number of Participants With Respiratory Failure or Death Due to Respiratory Distress Syndrome (RDS)
Time frame: 72 hours
Number of participants who had respiratory failure due to RDS or death due to RDS; known as nasal continuous positive airway pressure (nCPAP) failure
5 secondary outcomes
Incidence of Respiratory Failure or Death Due to RDS
Time frame: 72 hours
Time to nCPAP Failure
Time frame: 72 hours
Incidence of Respiratory Failure or Death Due to RDS With Poisson Distribution Modeling
Time frame: 72 hours
Incidence of Respiratory Failure or Death Due to RDS
Time frame: 28 days
Number of Participants With Bronchopulmonary Dysplasia (BPD)
Time frame: 36 weeks post-menstrual age (PMA)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven Simonson, MD study director
Windtree Therapeutics
54 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lucinactant delivered via investigational delivery device and nCPAP in people with Respiratory Distress Syndrome, run by Windtree Therapeutics at 54 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 54 sites in United States, Poland, Chile, Canada, Hungary and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Respiratory Failure or Death Due to Respiratory Distress Syndrome (RDS) (time frame: 72 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2021.