SURFAXIN® Treatment for Prevention of Bronchopulmonary Dysplasia (BPD) in Very Low Birth Weight (VLBW) Infants.

• ClinicalTrials.gov Identifier: NCT00215540
• Recruitment Status: Terminated
• First Posted: September 22, 2005
• Results First Posted: June 13, 2012
• Last Update Posted: June 13, 2012
• Sponsor: Windtree Therapeutics
• Information provided by (Responsible Party): Windtree Therapeutics

Study Description

Brief 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 distress syndrome (RDS).

Condition or disease	Intervention/treatment	Phase
Respiratory Distress Syndrome, Newborn; Premature Birth; Bronchopulmonary Dysplasia	Drug: Lucinactant 175 mg/kg; Drug: Lucinactant 90 mg/kg; Drug: Placebo	Phase 2

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

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 136 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Prevention
• 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
• Study Start Date: February 2005
• Actual Primary Completion Date: July 2006
• Actual Study Completion Date: July 2006

Arms and Interventions

Arm	Intervention/treatment
Experimental: SURFAXIN High Dose; SURFAXIN (lucinactant) at 175 mg/kg	Drug: Lucinactant 175 mg/kg; 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).; Other Names: SURFAXIN; Lucinactant; Surfactant
Experimental: SURFAXIN Low Dose; SURFAXIN (lucinactant) at 90 mg/kg	Drug: Lucinactant 90 mg/kg; 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).; Other Names: SURFAXIN; Lucinactant; Surfactant
Placebo Comparator: Placebo; Sham air using 3.0 mL/kg volume of air	Drug: Placebo; 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).; Other Name: Sham Air

Outcome Measures

Primary Outcome Measures :

1. 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
2. All-cause Mortality [ Time Frame: 36 weeks PMA ]

Secondary Outcome Measures :

1. BPD at 28 Days [ Time Frame: 28 days of life ]
   BPD at 28 days of life, as determined by the need for supplemental oxygen
2. BPD at 36 Weeks [ Time Frame: 36 weeks PMA ]
   BPD at 36 weeks PMA as determined by the need for supplemental oxygen
3. Days Receiving Mechanical Ventilation (MV) [ Time Frame: 36 weeks PMA ]
   Number of days receiving mechanical ventilation
4. Duration of Supplemental Oxygen [ Time Frame: 36 weeks PMA ]
   Number of days receiving supplemental oxygen through 36 weeks PMA
5. 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 ]
   AUC for FiO₂calculated using the trapezoidal rule. Missing data imputed using last observation carried forward
6. 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 ]
   AUC for MAP (in mm Hg) calculated using the trapezoidal rule. Missing data imputed using last observation carried forward
7. Incidence of Death or BPD at 28 Days [ Time Frame: 28 days of life ]
   Death or BPD, defined as oxygen requirement at 28 days of life
8. Days in Hospital [ Time Frame: 36 weeks PMA ]
   The number of days spent in the hospital through 36 weeks PMA

Eligibility Criteria

• Ages Eligible for Study: 3 Days to 10 Days (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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
• 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

Contacts and Locations

Locations

United States, Pennsylvania

Discovery Laboratories, Inc.

Warrington, Pennsylvania, United States, 18976-3646

Sponsors and Collaborators

Windtree Therapeutics

Investigators

• Study Director: Carlos Guardia, MD; Windtree Therapeutics
• Principal Investigator: Matthew M Laughon, MD, MPH; University of North Carolina, Chapel Hill

More Information

Publications of Results:

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. Erratum In: Pediatrics. 2009 May;123(5):1436.

• Responsible Party: Windtree Therapeutics
• ClinicalTrials.gov Identifier: NCT00215540
• Other Study ID Numbers: KL4-BPD-01
• First Posted: September 22, 2005
• Results First Posted: June 13, 2012
• Last Update Posted: June 13, 2012
• Last Verified: May 2012

Keywords provided by Windtree Therapeutics:

Double-blind; Low Birth Weight; Surfactant; Placebo-Controlled; Premature Birth

Additional relevant MeSH terms:

Respiratory Distress Syndrome; Respiratory Distress Syndrome, Newborn; Bronchopulmonary Dysplasia; Premature Birth; Birth Weight; Obstetric Labor, Premature; Obstetric Labor Complications; Pregnancy Complications; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Infant, Premature, Diseases; Infant, Newborn, Diseases; Body Weight; Ventilator-Induced Lung Injury; Lung Injury; Pulmonary Surfactants; Respiratory System Agents