Respiratory Distress SyndromePhase 2NCT02528318
Trial To Assess The Safety And Tolerability Of Lucinactant For Inhalation In Preterm Neonates 26 to 28 Weeks PMA
A Phase 2 study testing Lucinactant for inhalation and nCPAP alone in people with Respiratory Distress Syndrome, run by Windtree Therapeutics at 20 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 26 to 28 weeks
- Conditions the study is for
- Respiratory Distress Syndrome
- What is being tested?
- Lucinactant for inhalation and nCPAP alone
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 20 sitesin 4 countries
- Study dates
- Aug 2015to 11 Aug 2017
- 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 28 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed consent from a legally authorized representative.
- Gestational age 26 to 28 completed weeks post menstrual age (PMA).
- Successful implementation of controlled nCPAP within 90 minutes after birth.
- Spontaneous breathing.
- Chest radiograph consistent with RDS.
- 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.
Who cannot join
- Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth.
- 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.
- A 5 minute Apgar score < 5.
- Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth.
- Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH).
- Known or suspected chromosomal abnormality or syndrome.
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- Premature rupture of membranes (PROM) > 2 weeks.
- Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.
- Need for endotracheal intubation and mechanical ventilation.
- Has been administered: another investigational agent or investigational medical device, any other surfactant agent, steroid treatment after birth.
Trial locations and contacts
20 sites in 4 countries.
20 sites in 4 countries
United States10 sites
Loma Linda University Medical Center
Loma Linda, California, 92354
University of Louisville
Louisville, Kentucky, 40202
University of Miami
Miami, Florida, 33136
Columbia University College of Physicians and Surgeons, New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, 10032
Christiana Care Health System
Newark, Delaware, 19713
University of Nebraska Medical Center
Omaha, Nebraska, 68105
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
Canada4 sites
Foothills Medical Centre
Calgary, Alberta, T2N 2T9
Royal Alexandria Hospital
Edmonton, Alberta, T5H 3V9
Montreal Children's Hospital
Montreal, Quebec, H4A 3J1
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Poland4 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
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
Szpital Kliniczny im. Ks, Anny Mazowieckiej Klinika Neonatologii
Warsaw, Masovian Voivodeship, 00-315
Chile2 sites
Hospital Dr Sotero Del Rio
Santiago, 8207257
Hospital San Juan de Dios
Santiago, 8350488
About this study
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
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.
Read the 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.
- Conditions
- Respiratory Distress Syndrome
- Being tested
- Lucinactant for inhalation, nCPAP alone
- 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 for inhalationCombination product
Also called AEROSURF®
Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)
nCPAP aloneDevice
Also called nasal continuous positive airway pressure
nCPAP therapy
| Study group | Role | What they receive |
|---|---|---|
| 50 mg/kg | Experimental | Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP
1 repeat dose allowed if repeat dosing criteria are met. Receives: Combination Product: Lucinactant for inhalation |
| 75 mg/kg | Experimental | Lucinactant for inhalation 75 mg TPL/kg with nCPAP
1 repeat dose allowed if repeat dosing criteria are met. Receives: Combination Product: Lucinactant for inhalation |
| 100 mg/kg | Experimental | Lucinactant for inhalation 100 mg TPL/kg with nCPAP
1 repeat dose allowed if repeat dosing criteria are met. Receives: Combination Product: Lucinactant for inhalation |
| 150 mg/kg | Experimental | Lucinactant for inhalation 150 mg TPL/kg with nCPAP
1 repeat dose will be allowed if repeat dosing criteria are met. Receives: Combination Product: Lucinactant for inhalation |
| nCPAP alone | Active comparator | nCPAP therapy alone Receives: Device: nCPAP alone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Peri-Dosing Adverse Events - Initial Dose
Number of Participants with adverse events that were experienced during the initial study treatment
Measured: Randomization to 24 Hours Post Randomization
Number of Participants With Air Leak
Number of participants with air leak (includes pneumothorax, pulmonary interstitial emphysema (PIE), pneumomediastinum, pneumopericardium, subcutaneous emphysema)
Measured: 7 days
Secondary outcomes (6)
Number of Participants With Worsening of Respiratory Status Criteria
Number of participants with worsening in one of 12 respiratory status criteria through 72 hours post randomization (need for additional surfactant therapy, desaturation < 80%, heart rate < 100 bpm, sustained fraction of inspired oxygen (FiO2) > 0.50, arterial carbon dioxide (PCO2) > 65 mmHg, sustained apnea, persistent arterial pH < 7.2, intubation for any reason, nCPAP > 7 cmH2O, initiation of intermittent positive pressure ventilation, death, principal investigator determination of worsening status)
Measured: Randomization to 72 Hours Post Randomization
Bronchopulmonary Dysplasia
Number of participants with bronchopulmonary dysplasia (BPD) and number of participants alive and without BPD at 36 weeks post-menstrual age (PMA)
Measured: Randomization to 36 weeks PMA
Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure
Participants who required intubation for mechanical ventilation or surfactant administration were defined as having failed nCPAP
Measured: Randomization to 72 Hours Post Randomization
Death
Number of participants who died during the study
Measured: Randomization to 36 weeks PMA
FiO2
Observed and change from baseline measurements for fraction of inspired oxygen (FiO2). Values represent the amount (fraction) of oxygen in the air the participant inspires; the values themselves do not have units. The normal amount of oxygen in air ("room air") is 21%, or 0.21.
Measured: Randomization to 72 hours post randomization
Number of Participants With Complications of Prematurity
Number of participants with pre-specified common complications of prematurity.
Measured: Randomization to 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 posted19 Aug 2015 (estimated)
- Study startAug 2015
- Primary completion31 May 2017
- Study completion11 Aug 2017
Why it stopped: Sponsor decision to terminate after 3 dosing groups
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
- Sequential
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Windtree Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Steve Simonson, MD, Study director, Windtree Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02528318
- Sponsor's protocol ID
- 03-CL-1401
- Status last verified
- Jul 2019
- First submitted
- 17 Aug 2015
- Last updated
- 23 Jul 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02528318
- Acronym / sponsor ID
- 03-CL-1401
- Conditions
- Respiratory Distress Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision to terminate after 3 dosing groups
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Windtree TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Aug 2015 (actual)
- Primary completion
- 31 May 2017 (actual)
- Study completion
- 11 Aug 2017 (actual)
- First posted
- 19 Aug 2015 (estimated)
- Results
- Posted 23 Jul 2019
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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 50 mg/kg | Experimental | Combination Product: Lucinactant for inhalationLucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met. |
| 75 mg/kg | Experimental | Combination Product: Lucinactant for inhalationLucinactant for inhalation 75 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met. |
| 100 mg/kg | Experimental | Combination Product: Lucinactant for inhalationLucinactant for inhalation 100 mg TPL/kg with nCPAP 1 repeat dose allowed if repeat dosing criteria are met. |
| 150 mg/kg | Experimental | Combination Product: Lucinactant for inhalationLucinactant for inhalation 150 mg TPL/kg with nCPAP 1 repeat dose will be allowed if repeat dosing criteria are met. |
| nCPAP alone | Active comparator | Device: nCPAP alonenCPAP therapy alone |
Who may qualify
- Ages
- 26 to 28 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Informed consent from a legally authorized representative.
- Gestational age 26 to 28 completed weeks post menstrual age (PMA).
- Successful implementation of controlled nCPAP within 90 minutes after birth.
- Spontaneous breathing.
- Chest radiograph consistent with RDS.
Key exclusion criteria (10 listed)
- Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth.
- 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.
- A 5 minute Apgar score < 5.
- Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth.
- Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH).
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: AEROSURF®Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product) | Combination product | 100 mg/kg, 150 mg/kg, 50 mg/kg, 75 mg/kg |
| nCPAP aloneAlso: nasal continuous positive airway pressurenCPAP therapy | Device | nCPAP alone |
Primary outcome
Number of Participants With Peri-Dosing Adverse Events - Initial Dose
Time frame: Randomization to 24 Hours Post Randomization
Number of Participants with adverse events that were experienced during the initial study treatment
Number of Participants With Air Leak
Time frame: 7 days
Number of participants with air leak (includes pneumothorax, pulmonary interstitial emphysema (PIE), pneumomediastinum, pneumopericardium, subcutaneous emphysema)
6 secondary outcomes
Number of Participants With Worsening of Respiratory Status Criteria
Time frame: Randomization to 72 Hours Post Randomization
Bronchopulmonary Dysplasia
Time frame: Randomization to 36 weeks PMA
Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure
Time frame: Randomization to 72 Hours Post Randomization
Death
Time frame: Randomization to 36 weeks PMA
FiO2
Time frame: Randomization to 72 hours post randomization
Number of Participants With Complications of Prematurity
Time frame: Randomization to 36 weeks PMA
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2019.
- 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.
Steve Simonson, MD study director
Windtree Therapeutics
20 sites in 4 countries: 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 alone in people with Respiratory Distress Syndrome, run by Windtree Therapeutics at 20 sites in 4 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 10 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 23 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, Canada, Poland, Chile. 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 Jul 2019). 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 Peri-Dosing Adverse Events - Initial Dose (time frame: Randomization to 24 Hours Post Randomization). The study lists 2 primary outcomes in all. 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 Jul 2019.