Respiratory Distress Syndrome, NewbornPhase 2NCT04264156
A Safety and Efficacy Study of Lucinactant for Inhalation in Preterm Neonates 26 to 32 Weeks Gestational Age
A Phase 2 study testing Lucinactant for Inhalation and nCPAP Only in people with Respiratory Distress Syndrome, Newborn, run by Windtree Therapeutics at 3 sites in Poland.
Trial 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. Read more about this study
- Who is it for?
- Ages 30 minutes to 6 hours
- Conditions the study is for
- Respiratory Distress Syndrome, Newborn
- What is being tested?
- Lucinactant for Inhalation and nCPAP Only
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 3 sitesPoland
- Study dates
- 18 Apr 2020to 28 Mar 2021
- 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 30 minutes to 6 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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%.
Who cannot join
- 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.
Show all 11Show fewer
- 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.
Trial locations and contacts
3 sites in 1 country.
Poland3 sites
Szpital Specjalistyczny nr 2 w Bytomiu Oddzial Noworodkow Blok Va
Bytom, 41-902
Ginekologiczno-Polozniczy Szpital Kliniczny Uniwewersytetu Medycznego im. Karola Marcinkowskiego
Poznan, 60-535
Samodzielny Publiczny Spcjalistyczny Zaklad Opieki Zdrowotnej "Zdroje", Oddzial Noworodkow
Szczecin, 70-780
About this study
Official title: A Multinational, Multicenter, Masked, Randomized, Controlled Study to Assess the Safety and Efficacy of Lucinactant for Inhalation Versus nCPAP Alone in Preterm Neonates 26 to 32 Weeks Gestational Age With RDS
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).
Read the detailed description
An unmet medical need exists for a means to deliver surfactant replacement therapy (SRT) to preterm neonates with RDS supported with nCPAP early in the course of the disease. This strategy has the potential to improve RDS prior to the development of respiratory failure, thereby avoiding the need for endotracheal intubation and mechanical ventilation (MV), or reduce the duration of MV, and the resultant potential for morbidity and complications. The ability to administer SRT via aerosol has the potential to address this unmet need.
Lucinactant for inhalation (AEROSURF) is an investigational drug-device combination product, designed to deliver aerosolized SRT to preterm neonates with RDS who are being supported with nCPAP. The drug component of lucinactant for inhalation is lyophilized lucinactant, a lyophilized form of SURFAXIN® (lucinactant) Intratracheal Suspension. The device component, the AEROSURF Delivery System (ADS), the next-generation device following use of the prototype device in earlier trials, uses novel technology to aerosolize lucinactant for inhalation.
This study evaluates 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 pre-specified outcome measures. In addition, this study will evaluate the device and the ability to administer up to 3 repeat doses.
- Conditions
- Respiratory Distress Syndrome, Newborn
- Being tested
- Lucinactant for Inhalation, nCPAP Only
- Keywords
- intubation, surfactant, continuous positive airway pressure, aerosol, respiratory distress syndrome
- Medical subject headings
- Respiratory Distress Syndrome, Newborn, Respiratory Distress Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lucinactant for InhalationCombination product
Also called AEROSURF
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).
nCPAP OnlyOther
Nasal continuous positive airway pressure (nCPAP) alone
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Other: nCPAP Only |
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
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
Measured: 28 days of life
Secondary outcomes (3)
Number With BPD
Number of participants with bronchopulmonary dysplasia (BPD)
Measured: 36 weeks post-menstrual age (PMA)
Mortality
All-cause mortality
Measured: 36 weeks PMA or 28 days of life (whichever is later)
Number of Participants With Common Complications of Prematurity
Number of participants with complications including intraventricular hemorrhage, periventricular leukomalacia, pulmonary hemorrhage, apnea, necrotizing enterocolitis, patent ductus arteriosus, acquired sepsis, and retinopathy of prematurity.
Measured: 36 weeks PMA
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2020
- Study start18 Apr 2020
- Primary completion31 Jan 2021
- Study completion28 Mar 2021
Why it stopped: Corporate business reasons
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 Syndrome, NewbornStudies 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
- Model description
- 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).
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Windtree Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carlos Guardia, MD, Principal investigator, Windtree Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04264156
- Sponsor's protocol ID
- 03-CL-1702
- Status last verified
- Apr 2023
- First submitted
- 7 Feb 2020
- Last updated
- 24 May 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2023. 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 Versus nCPAP Alone in Preterm Neonates 26 to 32 Weeks Gestational Age With RDS
- ClinicalTrials.gov ID
- NCT04264156
- Acronym / sponsor ID
- 03-CL-1702
- Conditions
- Respiratory Distress Syndrome, Newborn
- Keywords
- intubation, surfactant, continuous positive airway pressure, aerosol, respiratory distress syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Corporate business reasons
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Windtree TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 18 Apr 2020 (actual)
- Primary completion
- 31 Jan 2021 (actual)
- Study completion
- 28 Mar 2021 (actual)
- First posted
- 11 Feb 2020 (actual)
- Results
- Posted 24 May 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
An unmet medical need exists for a means to deliver surfactant replacement therapy (SRT) to preterm neonates with RDS supported with nCPAP early in the course of the disease. This strategy has the potential to improve RDS prior to the development of respiratory failure, thereby avoiding the need for endotracheal intubation and mechanical ventilation (MV), or reduce the duration of MV, and the resultant potential for morbidity and complications. The ability to administer SRT via aerosol has the potential to address this unmet need.
Lucinactant for inhalation (AEROSURF) is an investigational drug-device combination product, designed to deliver aerosolized SRT to preterm neonates with RDS who are being supported with nCPAP. The drug component of lucinactant for inhalation is lyophilized lucinactant, a lyophilized form of SURFAXIN® (lucinactant) Intratracheal Suspension. The device component, the AEROSURF Delivery System (ADS), the next-generation device following use of the prototype device in earlier trials, uses novel technology to aerosolize lucinactant for inhalation.
This study evaluates 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 pre-specified outcome measures. In addition, this study will evaluate the device and the ability to administer up to 3 repeat doses.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- 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).
| Arm | Type | Receives |
|---|---|---|
| Lucinactant (160 mg/kg) + nCPAP | Experimental | Combination Product: Lucinactant for InhalationLucinactant 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 |
| nCPAP Only | Sham comparator | Other: nCPAP OnlynCPAP Only as sham comparator. Bubble nCPAP is standard of care. Treatment time behind barrier to match active treatment delivery time |
Who may qualify
- Ages
- 30 minutes to 6 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
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 for InhalationAlso: AEROSURFA drug-device combination product that delivers aerosolized SRT to preterm neonates with RDS who are being supported with nasal continuous positive airway pressure (nCPAP). | Combination product | Lucinactant (160 mg/kg) + nCPAP |
| nCPAP OnlyNasal continuous positive airway pressure (nCPAP) alone | Other | nCPAP Only |
Primary outcome
Number of Participants With Respiratory Failure or Death
Time frame: 28 days of life
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
3 secondary outcomes
Number With BPD
Time frame: 36 weeks post-menstrual age (PMA)
Mortality
Time frame: 36 weeks PMA or 28 days of life (whichever is later)
Number of Participants With Common Complications of Prematurity
Time frame: 36 weeks PMA
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Carlos Guardia, MD principal investigator
Windtree Therapeutics, Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lucinactant for Inhalation and nCPAP Only in people with Respiratory Distress Syndrome, Newborn, run by Windtree Therapeutics at 3 sites in Poland.
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 11 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: stopped early, as of the sponsor's last update on 24 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Poland. 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. Some participants in this study may receive a placebo. 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 24 May 2023). 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 (time frame: 28 days of life). 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 24 May 2023.