Respiratory Distress Syndrome, NewbornPhase 2NCT00215540
SURFAXIN® Treatment for Prevention of Bronchopulmonary Dysplasia (BPD) in Very Low Birth Weight (VLBW) Infants.
A Phase 2 study testing Lucinactant 175 mg/kg and Lucinactant 90 mg/kg in people with Respiratory Distress Syndrome, Newborn and Premature Birth, run by Windtree Therapeutics at 1 site in United States.
Trial summary
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required continued intubation and received surfactants for the prevention or treatment of respiratory… Read more about this study
- Who is it for?
- Ages 3 to 10 days
- Conditions the study is for
- Respiratory Distress Syndrome, Newborn, Premature Birth, Bronchopulmonary Dysplasia
- What is being tested?
- Lucinactant 175 mg/kg and Lucinactant 90 mg/kg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 136 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2005to Jul 2006
- 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 3 to 10 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Premature infants between 600 and 900 grams birth weight
- Intubated and on mechanical ventilation
- Sustained (>= 30 minutes) fraction of inspired oxygen (FiO₂) >= 0.30 within 8 hours prior to randomization
Who cannot join
- Mother has prolonged rupture of membranes ≥ 2 weeks
- Culture-proven sepsis
- High grade intraventricular hemorrhage (IVH)
- Congenital heart disease
- Congential anomalies inconsistent with life or likely to confound efficacy or safety endpoints
- FiO₂≥ 0.80 and mean airway pressure (MAP) ≥ 12 cmH2O at day of life (DOL) 3
Show all 11Show fewer
- FiO₂< 0.25 at any time between meeting the entry criteria to immediately prior to randomization
- Concomitant use of any other surfactant within the first 48 hours of life
- Prior use of nitric oxide
- Prior use of steroids
- Current participation in any other clinical trial or has received an experimental drug or used an experimental device
Trial locations and contacts
1 site in 1 country.
United States1 site
Discovery Laboratories, Inc.
Warrington, Pennsylvania, 18976-3646
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Safety and Efficacy of SURFAXIN® (Lucinactant), in Very Low Birth Weight (VLBW) Infants at Risk for Developing Bronchopulmonary Dysplasia
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required continued intubation and received surfactants for the prevention or treatment of respiratory distress syndrome (RDS).
Read the detailed description
Determine the safety and tolerability of SURFAXIN administration in the first weeks of life as a therapeutic approach for prevention of BPD. Determine whether treatment with SURFAXIN during the first two to three weeks of life can decrease the proportion of infants on mechanical ventilation or oxygen or the incidence of death or BPD in VLBW infants when assessed at 28 days of life and 36 weeks post-menstrual age (as determined by the need for supplemental oxygen).
- Being tested
- Lucinactant 175 mg/kg, Lucinactant 90 mg/kg, Placebo
- Keywords
- Double-blind, Low Birth Weight, Surfactant, Placebo-Controlled, Premature Birth
- Medical subject headings
- Respiratory Distress Syndrome, Newborn, Premature Birth, Bronchopulmonary Dysplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lucinactant 175 mg/kgDrug
Also called SURFAXIN, Lucinactant, Surfactant
Administered via slow intra-tracheal instillation at a dose of 175 mg/kg (5.8 mL/kg of a 30-mg/mL suspension). Initial treatment given no later than 1 hour after randomization. Additional treatments were administered every 48 hours up to a maximum of 5 doses (up to day of life (DOL) 18).
Lucinactant 90 mg/kgDrug
Also called SURFAXIN, Lucinactant, Surfactant
Administered via slow intra-tracheal instillation at a dose of 90 mg/kg (3.0 mL/kg of a 30-mg/mL suspension). Initial treatment given no later than 1 hour after randomization. Additional treatments were administered every 48 hours up to a maximum of 5 doses (up to DOL 18).
PlaceboDrug
Also called Sham Air
Sham air was administered via slow intratracheal instillation at a dose of 3.0 mL/kg volume of air. The initial treatment was given no later than 1 hour after randomization. Additional treatment were administered every 48 hours up to a maximum of 5 doses (up to DOL 18).
| Study group | Role | What they receive |
|---|---|---|
| SURFAXIN High Dose | Experimental | SURFAXIN (lucinactant) at 175 mg/kg Receives: Drug: Lucinactant 175 mg/kg |
| SURFAXIN Low Dose | Experimental | SURFAXIN (lucinactant) at 90 mg/kg Receives: Drug: Lucinactant 90 mg/kg |
| Placebo | Placebo comparator | Sham air using 3.0 mL/kg volume of air Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Death or Bronchopulmonary Dysplasia (BPD) at 36 Weeks
Number of participants who died or developed BPD, defined as oxygen requirement at 36 Weeks post-menstrual age
Measured: 36 weeks post-menstrual age (PMA)
All-cause Mortality
Measured: 36 weeks PMA
Secondary outcomes (8)
BPD at 28 Days
BPD at 28 days of life, as determined by the need for supplemental oxygen
Measured: 28 days of life
BPD at 36 Weeks
BPD at 36 weeks PMA as determined by the need for supplemental oxygen
Measured: 36 weeks PMA
Days Receiving Mechanical Ventilation (MV)
Number of days receiving mechanical ventilation
Measured: 36 weeks PMA
Duration of Supplemental Oxygen
Number of days receiving supplemental oxygen through 36 weeks PMA
Measured: 36 weeks PMA
Area Under the Curve for Fraction of Inspired Oxygen (FiO₂)
AUC for FiO₂calculated using the trapezoidal rule. Missing data imputed using last observation carried forward
Measured: 15 minutes prior to dose 1, 2 hours post dose 1, 6 hours post dose 1, 24 hours post dose 1, and daily from Study Day 2 to Study Day 25 and Day of Life 28
Area Under the Curve for Mean Arterial Pressure (MAP)
AUC for MAP (in mm Hg) calculated using the trapezoidal rule. Missing data imputed using last observation carried forward
Measured: 15 minutes prior to dose 1, 2 hours post dose 1, 6 hours post dose 1, 24 hours post dose 1, and daily from Study Day 2 to Study Day 25, and day of life 28
Incidence of Death or BPD at 28 Days
Death or BPD, defined as oxygen requirement at 28 days of life
Measured: 28 days of life
Days in Hospital
The number of days spent in the hospital through 36 weeks PMA
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 posted22 Sep 2005 (estimated)
- Study startFeb 2005
- Primary completionJul 2006
- Study completionJul 2006
Why it stopped: Concerns related to availability of drug product
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 136 participants (actual)
Sponsor and investigators
- Lead sponsor
- Windtree Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carlos Guardia, MD, Study director, Windtree TherapeuticsMatthew M Laughon, MD, MPH, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00215540
- Sponsor's protocol ID
- KL4-BPD-01
- Status last verified
- May 2012
- First submitted
- 14 Sep 2005
- Last updated
- 13 Jun 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Laughon M, Bose C, Moya F, Aschner J, Donn SM, Morabito C, Cummings JJ, Segal R, Guardia C, Liu G; Surfaxin Study Group. A pilot randomized, controlled trial of later treatment with a peptide-containing, synthetic surfactant for the prevention of bronchopulmonary dysplasia. Pediatrics. 2009 Jan;123(1):89-96. doi: 10.1542/peds.2007-2680.
PubMed 19117865
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Safety and Efficacy of SURFAXIN® (Lucinactant), in Very Low Birth Weight (VLBW) Infants at Risk for Developing Bronchopulmonary Dysplasia
- ClinicalTrials.gov ID
- NCT00215540
- Acronym / sponsor ID
- KL4-BPD-01
- Keywords
- Double-blind, Low Birth Weight, Surfactant, Placebo-Controlled, Premature Birth
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Concerns related to availability of drug product
- Enrolment
- 136 participants (actual)
- Lead sponsor
- Windtree TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Feb 2005
- Primary completion
- Jul 2006 (actual)
- Study completion
- Jul 2006 (actual)
- First posted
- 22 Sep 2005 (estimated)
- Results
- Posted 13 Jun 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required continued intubation and received surfactants for the prevention or treatment of respiratory distress syndrome (RDS).
Detailed description
Determine the safety and tolerability of SURFAXIN administration in the first weeks of life as a therapeutic approach for prevention of BPD. Determine whether treatment with SURFAXIN during the first two to three weeks of life can decrease the proportion of infants on mechanical ventilation or oxygen or the incidence of death or BPD in VLBW infants when assessed at 28 days of life and 36 weeks post-menstrual age (as determined by the need for supplemental oxygen).
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SURFAXIN High Dose | Experimental | Drug: Lucinactant 175 mg/kgSURFAXIN (lucinactant) at 175 mg/kg |
| SURFAXIN Low Dose | Experimental | Drug: Lucinactant 90 mg/kgSURFAXIN (lucinactant) at 90 mg/kg |
| Placebo | Placebo comparator | Drug: PlaceboSham air using 3.0 mL/kg volume of air |
Who may qualify
- Ages
- 3 to 10 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Premature infants between 600 and 900 grams birth weight
- Intubated and on mechanical ventilation
- Sustained (>= 30 minutes) fraction of inspired oxygen (FiO₂) >= 0.30 within 8 hours prior to randomization
Key exclusion criteria (11 listed)
- Mother has prolonged rupture of membranes ≥ 2 weeks
- Culture-proven sepsis
- High grade intraventricular hemorrhage (IVH)
- Congenital heart disease
- Congential anomalies inconsistent with life or likely to confound efficacy or safety endpoints
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 175 mg/kgAlso: SURFAXIN, Lucinactant, SurfactantAdministered via slow intra-tracheal instillation at a dose of 175 mg/kg (5.8 mL/kg of a 30-mg/mL suspension). Initial treatment given no later than 1 hour after randomization. Additional treatments were administered every 48 hours up to a maximum of 5 doses (up to day of life (DOL) 18). | Drug | SURFAXIN High Dose |
| Lucinactant 90 mg/kgAlso: SURFAXIN, Lucinactant, SurfactantAdministered via slow intra-tracheal instillation at a dose of 90 mg/kg (3.0 mL/kg of a 30-mg/mL suspension). Initial treatment given no later than 1 hour after randomization. Additional treatments were administered every 48 hours up to a maximum of 5 doses (up to DOL 18). | Drug | SURFAXIN Low Dose |
| PlaceboAlso: Sham AirSham air was administered via slow intratracheal instillation at a dose of 3.0 mL/kg volume of air. The initial treatment was given no later than 1 hour after randomization. Additional treatment were administered every 48 hours up to a maximum of 5 doses (up to DOL 18). | Drug | Placebo |
Primary outcome
Incidence of Death or Bronchopulmonary Dysplasia (BPD) at 36 Weeks
Time frame: 36 weeks post-menstrual age (PMA)
Number of participants who died or developed BPD, defined as oxygen requirement at 36 Weeks post-menstrual age
All-cause Mortality
Time frame: 36 weeks PMA
8 secondary outcomes
BPD at 28 Days
Time frame: 28 days of life
BPD at 36 Weeks
Time frame: 36 weeks PMA
Days Receiving Mechanical Ventilation (MV)
Time frame: 36 weeks PMA
Duration of Supplemental Oxygen
Time frame: 36 weeks PMA
Area Under the Curve for Fraction of Inspired Oxygen (FiO₂)
Time frame: 15 minutes prior to dose 1, 2 hours post dose 1, 6 hours post dose 1, 24 hours post dose 1, and daily from Study Day 2 to Study Day 25 and Day of Life 28
Area Under the Curve for Mean Arterial Pressure (MAP)
Time frame: 15 minutes prior to dose 1, 2 hours post dose 1, 6 hours post dose 1, 24 hours post dose 1, and daily from Study Day 2 to Study Day 25, and day of life 28
Incidence of Death or BPD at 28 Days
Time frame: 28 days of life
Days in Hospital
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 13 Jun 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 19117865Laughon M, Bose C, Moya F, Aschner J, Donn SM, Morabito C, Cummings JJ, Segal R, Guardia C, Liu G; Surfaxin Study Group. A pilot randomized, controlled trial of later treatment with a peptide-containing, synthetic surfactant for the prevention of bronchopulmonary dysplasia. Pediatrics. 2009 Jan;123(1):89-96. doi: 10.1542/peds.2007-2680.
Related trials for Respiratory Distress Syndrome, Newborn
Enrolling by invitation
Personalized Ventilator Settings for Patients on ECMO
Acute Respiratory Distress Syndrome (ARDS), Extracorporeal Membrane Oxygenation
Not yet recruitingObservational study
Development and Multicenter Validation of an Explainable System for Neonatal ARDS-Compatible Phenotyping
Acute Respiratory Distress Syndrome
RecruitingObservational study
Assessment of Blood Viscosity in ARDS (Acute Respiratory Distress Syndrome)
ARDS
Recruiting
Comparing Surfactant Administration Through Supraglottic Airway and Thin Catheter for Preterm Infants
Surfactant, Respiratory Distress Syndrome (Neonatal)
Recruiting
REspiratory MEchanics for Delivering Individualised Exogenous Surfactant
Respiratory Distress Syndrome in Premature Infant
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 study director
Windtree Therapeutics
Matthew M Laughon, MD, MPH principal investigator
University of North Carolina, Chapel Hill
1 site 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 175 mg/kg and Lucinactant 90 mg/kg in people with Respiratory Distress Syndrome, Newborn and Premature Birth, run by Windtree Therapeutics at 1 site 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 3 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 13 Jun 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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 13 Jun 2012). 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?
Incidence of Death or Bronchopulmonary Dysplasia (BPD) at 36 Weeks (time frame: 36 weeks post-menstrual age (PMA)). 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?
On the ClinicalTrials.gov record for NCT00215540, which also lists 1 linked publication.
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 13 Jun 2012.