Respiratory Distress SyndromePhase 2NCT02074059

Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Premature Neonates

A Phase 2 study testing Lucinactant for Inhalation and nCPAP alone in people with Respiratory Distress Syndrome, run by Windtree Therapeutics at 11 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The primary objective of this study is to evaluate the safety and tolerability of aerosolized surfactant, specifically lucinactant for inhalation, administered in escalating inhaled doses to preterm neonates 29 to 34 weeks gestational age who are receiving nasal continuous positive airway pressure (nCPAP) for… Read more about this study

Who is it for?: Ages 29 to 34 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: 80 participants
Where: 11 sitesUnited States
Study dates: Feb 2014to Nov 2015
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 29 to 34 weeks
All sexes
No healthy volunteers
Child

Who may be able to join

Informed consent from a legally authorized representative
Gestational age 29 to 34 completed weeks (34 weeks 6 days) post menstrual age (PMA)
Successful implementation of controlled nCPAP within 90 minutes after birth
Spontaneous breathing
Chest radiograph consistent with RDS
Need for moderate levels of supplemental oxygen and nCPAP to maintain oxygen saturation of 88% to 95% for at least 30 minutes within the first 21 hours after birth

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

Show all 10Show fewer

Prolong 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 exposure to an investigational medical device, any other surfactant agent, steroid treatment after birth

Trial locations and contacts

11 sites in 1 country.

City, postcode or hospital

11 sites in 1 country

United States11 sites

University of Alabama at Birmingham
Birmingham, Alabama, 35249
Duke University Medical Center
Durham, North Carolina, 27710
Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Loma Linda University Medical Center
Loma Linda, California, 92354
University of Louisville
Louisville, Kentucky, 40202
University of Miami
Miami, Florida, 33136
Mid Atlantic Neonatology Associates
Morristown, New Jersey, 07960
Christiana Care Health System
Newark, Delaware, 19713
Providence St. Vincent Medical Center
Portland, Oregon, 97225
Sharp Mary Birch Hospital for Women and Newborns
San Diego, California, 92123

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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 29 to 34 Weeks PMA: Dose Escalation and Study Extension

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 InhalationDrug
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 groups in NCT02074059
Study group	Role	What they receive
Aerosolized lucinactant (25 mg/kg)	Experimental	25 mg total phospholipids (TPL)/kg: Lucinactant for inhalation with nCPAP Receives: Drug: Lucinactant for Inhalation
Aerosolized lucinactant (50 mg/kg)	Experimental	50 mg TPL/kg: Lucinactant for inhalation with nCPAP Receives: Drug: Lucinactant for Inhalation
Aerosolized lucinactant (75 mg/kg)	Experimental	75 mg TPL/kg: Lucinactant for inhalation with nCPAP Receives: Drug: Lucinactant for Inhalation
Aerosolized lucinactant (100 mg/kg)	Experimental	100 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met. Receives: Drug: Lucinactant for Inhalation
Aerosolized lucinactant (150 mg/kg)	Experimental	150 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met. Receives: Drug: 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.

Peri-Dosing Events
Pre-specified adverse events that occured during treatment administration; does not include nCPAP alone
Measured: Within 48 Hours after Initiation of Study Treatment
All Cause Mortality
Measured: Within 36 Weeks PMA
Oxygen Saturation Levels
Oxygen saturation as determined by pulse oximetry
Measured: Within 3 Hours of Randomization
Serum Electrolytes
Measured: 24 Hours Post Randomization

Secondary outcomes (1)

PCO2
Arterial carbon dioxide
Measured: Within 3 Hours of Randomization

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Feb 2014Nov 2015

First posted28 Feb 2014 (estimated)
Study startFeb 2014
Primary completionOct 2015
Study completionNov 2015

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
Model description: Sequential escalating dosing groups, each group contained an active and control arm of equal size.
Masking: None (open label)
Enrolment: 80 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02074059
Sponsor's protocol ID: 03-CL-1201
Other IDs: 4R44HL107000-02
Status last verified: Mar 2017
First submitted: 25 Feb 2014
Last updated: 21 Apr 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Recruiting
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Study at a glance

From the registry record, last updated by the sponsor on 21 Apr 2017. 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 29 to 34 Weeks PMA: Dose Escalation and Study Extension
ClinicalTrials.gov ID: NCT02074059
Acronym / sponsor ID: 03-CL-1201
Conditions: Respiratory Distress Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 80 participants (actual)
Lead sponsor: Windtree TherapeuticsIndustry (pharma, biotech, devices)
Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
Study start: Feb 2014
Primary completion: Oct 2015 (actual)
Study completion: Nov 2015 (actual)
First posted: 28 Feb 2014 (estimated)
Results: Posted 21 Apr 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 6
Model description: Sequential escalating dosing groups, each group contained an active and control arm of equal size.

Arm	Type	Receives
Aerosolized lucinactant (25 mg/kg)	Experimental	Drug: Lucinactant for Inhalation25 mg total phospholipids (TPL)/kg: Lucinactant for inhalation with nCPAP
Aerosolized lucinactant (50 mg/kg)	Experimental	Drug: Lucinactant for Inhalation50 mg TPL/kg: Lucinactant for inhalation with nCPAP
Aerosolized lucinactant (75 mg/kg)	Experimental	Drug: Lucinactant for Inhalation75 mg TPL/kg: Lucinactant for inhalation with nCPAP
Aerosolized lucinactant (100 mg/kg)	Experimental	Drug: Lucinactant for Inhalation100 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met.
Aerosolized lucinactant (150 mg/kg)	Experimental	Drug: Lucinactant for Inhalation150 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met.
nCPAP alone	Active comparator	Device: nCPAP alonenCPAP therapy alone

Who may qualify

Ages: 29 to 34 weeks
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (6 listed)

Informed consent from a legally authorized representative
Gestational age 29 to 34 completed weeks (34 weeks 6 days) 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: AerosurfLucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)	Drug	Aerosolized lucinactant (100 mg/kg), Aerosolized lucinactant (150 mg/kg), Aerosolized lucinactant (25 mg/kg), Aerosolized lucinactant (50 mg/kg), Aerosolized lucinactant (75 mg/kg)
nCPAP aloneAlso: nasal continuous positive airway pressurenCPAP therapy	Device	nCPAP alone

Primary outcome

Peri-Dosing Events
Time frame: Within 48 Hours after Initiation of Study Treatment
Pre-specified adverse events that occured during treatment administration; does not include nCPAP alone
All Cause Mortality
Time frame: Within 36 Weeks PMA
Oxygen Saturation Levels
Time frame: Within 3 Hours of Randomization
Oxygen saturation as determined by pulse oximetry
Serum Electrolytes
Time frame: 24 Hours Post Randomization

1 secondary outcome

PCO2
Time frame: Within 3 Hours of Randomization

Results and safety

Results posted to the registry on 21 Apr 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Aerolized Lucinactant (25 mg/kg)	8	8
Aerosolized Lucinactant (50 mg/kg)	8	8
Aerosolized Lucinactant (75 mg/kg)	8	8
Aerosolized Lucinactant (100 mg/kg)	8	8
Aerosolized Lucinactant (150 mg/kg)	8	7
nCPAP Alone	40	39

Primary outcome: Peri-Dosing Events

Time frame: Within 48 Hours after Initiation of Study Treatment. Number. Unit: participants

Measure	Aerolized Lucinactant (25 mg/kg) n = 8	Aerosolized Lucinactant (75 mg/kg) n = 8	Aerosolized Lucinactant (100 mg/kg) n = 8	nCPAP Alone n = 0
Bradycardia	0	0	1	–
Desaturation	2	1	2	–
Vomiting	0	1	1	–

Population analysed: Safety Population

Primary outcome: All Cause Mortality

Time frame: Within 36 Weeks PMA. Number. Unit: participants

Measure	Aerolized Lucinactant (25 mg/kg) n = 8	Aerosolized Lucinactant (50 mg/kg) n = 8	Aerosolized Lucinactant (75 mg/kg) n = 8	Aerosolized Lucinactant (100 mg/kg) n = 8	Aerosolized Lucinactant (150 mg/kg) n = 8	nCPAP Alone n = 40
Value	0	0	0	0	1	1

Population analysed: Safety Population

Primary outcome: Oxygen Saturation Levels

Time frame: Within 3 Hours of Randomization. Mean (Standard Deviation). Unit: percentage of oxygen saturation

Measure	Aerolized Lucinactant (25 mg/kg) n = 8	Aerosolized Lucinactant (50 mg/kg) n = 8	Aerosolized Lucinactant (75 mg/kg) n = 8	Aerosolized Lucinactant (100 mg/kg) n = 8	Aerosolized Lucinactant (150 mg/kg) n = 8	nCPAP Alone n = 40
At Randomization	94.0 (0)	91.3 (2.73)	96.4 (2.20)	93.9 (3.87)	92.5 (3.85)	93.3 (2.66)
At Initiation of Treatment	93.0 (4.00)	93.3 (1.37)	93.4 (4.24)	94.0 (3.82)	93.1 (1.25)	–
15 Minutes Post Radomization/Treatment	94.0 (0)	92.7 (4.68)	94.8 (5.15)	94.8 (3.62)	92.0 (4.66)	93.3 (3.39)
30 Minutes Post Randomization/Treatment	93.5 (2.12)	92.3 (2.25)	92.5 (5.95)	93.3 (4.83)	91.4 (3.89)	93.6 (3.33)
45 Minutes Post Randomization/Treatment	94.5 (0.71)	91.3 (3.88)	94.0 (4.72)	93.8 (3.11)	93.0 (2.27)	93.7 (3.04)
60 Minutes Post Randomization/Treatment	92.0 (0)	93.7 (2.07)	96.1 (2.47)	93.9 (3.72)	91.9 (3.00)	93.5 (3.68)
1.25 Hours Post Randomization/Treatment	94.0 (0)	93.2 (4.71)	95.6 (2.33)	94.0 (3.32)	92.0 (5.26)	93.9 (3.05)
1.5 Hours Post Randomization/Treatment	95.0 (4.24)	93.7 (3.27)	94.9 (3.52)	93.1 (6.34)	88.1 (16.98)	93.4 (2.83)

Population analysed: Safety Population

Primary outcome: Serum Electrolytes

Time frame: 24 Hours Post Randomization. Mean (Standard Deviation). Unit: mEq/L

Measure	Aerolized Lucinactant (25 mg/kg) n = 8	Aerosolized Lucinactant (50 mg/kg) n = 8	Aerosolized Lucinactant (High Dose) n = 8	Aerosolized Lucinactant (100 mg/kg) n = 8	Aerosolized Lucinactant (150 mg/kg) n = 8	nCPAP Alone n = 40
Sodium	140 (3.7)	137 (4.0)	139 (4.2)	140 (4.9)	137 (4.3)	139 (3.3)
Chloride	108 (3.0)	107 (4.2)	107 (6.6)	107 (6.3)	105 (4.5)	108 (3.5)
Potassium	3.86 (0.262)	4.66 (1.235)	3.86 (0.453)	4.46 (1.029)	4.09 (0.993)	4.47 (1.082)
Total Carbon Dioxide	20.8 (1.83)	21.3 (2.12)	22.0 (1.60)	24.5 (7.50)	21.9 (1.89)	21.7 (3.12)

Population analysed: All randomized subjects

1 secondary outcome is reported in the full results.

Adverse events

Time frame: From enrollment to 36 weeks post-menstrual age (PMA). As infants are enrolled at a gestational age of 29 to 34 weeks PMA, this represents a period of 2 to 7 weeks.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Aerolized Lucinactant (25 mg/kg)	2 of 8 (25%)	8 of 8 (100%)	Not reported
Aerosolized Lucinactant (50 mg/kg)	3 of 8 (37.5%)	8 of 8 (100%)	Not reported
Aerosolized Lucinactant (75 mg/kg)	2 of 8 (25%)	8 of 8 (100%)	Not reported
Aerosolized Lucinactant (100 mg/kg)	3 of 8 (37.5%)	7 of 8 (87.5%)	Not reported
Aerosolized Lucinactant (150 mg/kg)	3 of 8 (37.5%)	8 of 8 (100%)	Not reported
nCPAP Alone	7 of 40 (17.5%)	37 of 40 (92.5%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2017.
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.

Robert Segal, MD, FACP principal investigator

Windtree Therapeutics, Inc.

11 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 alone in people with Respiratory Distress Syndrome, run by Windtree Therapeutics at 11 sites in United States.

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: finished, as of the sponsor's last update on 21 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States. 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 21 Apr 2017). 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?

Peri-Dosing Events (time frame: Within 48 Hours after Initiation of Study Treatment). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02074059.

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 21 Apr 2017.