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

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).

Study Overview

• Status: Terminated
• Conditions: Premature Birth; Respiratory Distress Syndrome, Newborn; Bronchopulmonary Dysplasia
• Intervention / Treatment: Drug: Lucinactant 175 mg/kg; Drug: Lucinactant 90 mg/kg; Drug: Placebo

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 Type: Interventional
• Enrollment (Actual): 136
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Pennsylvania
    • Warrington, Pennsylvania, United States, 18976-3646
      • Discovery Laboratories, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 days to 1 week (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: PREVENTION
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

3

Arms and Interventions

Participant Group / 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 Names: Sham Air

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	36 weeks post-menstrual age (PMA)
All-cause Mortality		36 weeks PMA

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
BPD at 28 Days	BPD at 28 days of life, as determined by the need for supplemental oxygen	28 days of life
BPD at 36 Weeks	BPD at 36 weeks PMA as determined by the need for supplemental oxygen	36 weeks PMA
Days Receiving Mechanical Ventilation (MV)	Number of days receiving mechanical ventilation	36 weeks PMA
Duration of Supplemental Oxygen	Number of days receiving supplemental oxygen through 36 weeks PMA	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	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	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	28 days of life
Days in Hospital	The number of days spent in the hospital through 36 weeks PMA	36 weeks PMA

Collaborators and Investigators

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

Publications and helpful links

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

Study record dates

Study Major Dates

• Study Start: February 1, 2005
• Primary Completion (ACTUAL): July 1, 2006
• Study Completion (ACTUAL): July 1, 2006

Study Registration Dates

• First Submitted: September 14, 2005
• First Submitted That Met QC Criteria: September 21, 2005
• First Posted (ESTIMATE): September 22, 2005

Study Record Updates

• Last Update Posted (ESTIMATE): June 13, 2012
• Last Update Submitted That Met QC Criteria: May 11, 2012
• Last Verified: May 1, 2012

More Information

Terms related to this study

• Keywords: Double-blind; Surfactant; Placebo-Controlled; Premature Birth; Low Birth Weight
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Infant, Newborn, Diseases; Body Weight; Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Lung Injury; Infant, Premature, Diseases; Ventilator-Induced Lung Injury; Premature Birth; Birth Weight; Respiratory Distress Syndrome; Respiratory Distress Syndrome, Newborn; Bronchopulmonary Dysplasia; Respiratory System Agents; Pulmonary Surfactants
• Other Study ID Numbers: KL4-BPD-01