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Trial of Late Surfactant for Prevention of Bronchopulmonary Dysplasia

Phase 3
Completed
Conditions
Bronchopulmonary Dysplasia
Interventions
Drug: Sham (No Treatment)
Registration Number
NCT01022580
Lead Sponsor
Roberta Ballard
Brief Summary

The purpose of this study is to determine if late doses of Infasurf surfactant given when patients are receiving inhaled nitric oxide will interact to improve surfactant function and increase survival without BPD in treated infants.

Detailed Description

This is a multi-center, blinded, randomized controlled clinical trial to evaluate the effects of booster doses of exogenous surfactant (Infasurf®, calfactant) in addition to inhaled nitric oxide (iNO) on the outcome of survival without bronchopulmonary dysplasia (BPD, or chronic lung disease of prematurity, characterized by chronic lung dysfunction) at 36 weeks' post-menstrual age (PMA) in extremely low gestational age (ELGAN) infants that are at high risk of the development of BPD. This multi-center trial, with a planned enrollment of 524 infants, will also enable us to evaluate for any adverse effects of late surfactant treatment on short- and long-term outcomes, as we will be collecting data on effects of dosing of late surfactant, co-morbidities of prematurity and neurodevelopmental and pulmonary outcome at 1 year and 24 months corrected age. In addition, we will collect biological specimens for evaluation of the effects of late surfactant replacement therapy (administered as described in this trial) on surfactant function and inflammatory markers.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
511
Inclusion Criteria
  • <=28 0/7 weeks gestational age
  • Day of life 7-14
  • Intubated and mechanically ventilated
  • Plan to treat with inhaled nitric oxide
Exclusion Criteria
  • Serious congenital malformations or chromosomal abnormalities
  • Life expectancy <7 days from enrollment
  • Clinically unstable
  • Less tha 48 hours since last dose surfactant
  • Ability to obtain 36 week primary outcome information is unlikely

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Infasurf surfactant (ONY, Inc.)Infasurf surfactant (ONY, Inc.)Infants already receiving inhaled nitric oxide will receive scheduled doses of late surfactant (Infasurf) on study days 0, 2, 4, 6 and 8.
Sham (No Treatment)Sham (No Treatment)Infants already receiving inhaled nitric oxide will receive "Sham (no treatment)" doses on study days 0,2,4,6, and 8.
Primary Outcome Measures
NameTimeMethod
Survival Without BPD at 36 Weeks Post Menstrual Age.36 weeks post menstrual age +/- 1 week

BPD was diagnosed as need for respiratory support with either positive pressure and/or oxygen as evaluated by physiological oxygen/flow reduction.

Secondary Outcome Measures
NameTimeMethod
Respiratory Outcomes of TOLSURF Infants at 1 Year Corrected Age1 year

Evaluation of respiratory outcome through the first 12 months after birth. questionnaires were administered by phone at 3,6,9 and 12 months to determine respiratory resource use (medications, home support, and hospitalization) No Pulmonary Morbidity (NoPM) was reported for infants who had no resource use in any quarter and Persistent PM was reported for infants having resource use in 3 or 4 quarters.

Pulmonary Outcomes Through 2 Years of Age2 years

Evaluation of pulmonary outcome at 24 months of age. Evaluation of persistent wheezing based on reported wheezing during both first and second year of life.

Percentage of Participants With Neurodevelopmental Impairment (NDI) at 2 Years.2 years

At 24 months corrected age, children underwent neuropsychological testing with the Bayley Scales of Infant Development-Third Edition (Bayley-3), and composite cognitive, language, and motor scores were collected as well as subscale scores in receptive and expressive speech and fine and gross motor. Infants with greater than one impairment as identified by testing were classified as having neurodevelopmental impairment.

Survival Without BPD at 40 Weeks40 weeks PMA +/- 1 week

BPD was diagnosed as need for respiratory support with either positive pressure and/or oxygen as evaluated by physiological oxygen/flow reduction.

Trial Locations

Locations (21)

Alta Bates Medical Center

🇺🇸

Berkeley, California, United States

Stony Brook University Medical Center

🇺🇸

Stony Brook, New York, United States

Northwestern Memorial Hospital

🇺🇸

Chicago, Illinois, United States

UT Houston Health Science Center

🇺🇸

Houston, Texas, United States

Texas Children's Hospital

🇺🇸

Houston, Texas, United States

University of Washington, Seattle

🇺🇸

Seattle, Washington, United States

UT Memphis- Memphis Medical Center

🇺🇸

Memphis, Tennessee, United States

Childrens Hospital and Clinics of Minnesota- Minneapolis

🇺🇸

Minneapolis, Minnesota, United States

University of Minnesota Medical School

🇺🇸

Minneapolis, Minnesota, United States

University of Arkansas - Arkansas Childrens Hospital

🇺🇸

Little Rock, Arkansas, United States

Oakland Children's Hospital

🇺🇸

Oakland, California, United States

Florida Hospital for Children

🇺🇸

Orlando, Florida, United States

All Children's Hospital

🇺🇸

Saint Petersburg, Florida, United States

University of California, San Francisco

🇺🇸

San Francisco, California, United States

Wolfson Children's Hospital and Shands HospitaL

🇺🇸

Jacksonville, Florida, United States

Children's Memorial Hospital

🇺🇸

Chicago, Illinois, United States

Children's Mercy Hospital

🇺🇸

Kansas City, Missouri, United States

Children's Hospital and Clinics of Minnesota - St Paul

🇺🇸

Saint Paul, Minnesota, United States

Women's and Children's Hospital of Buffalo

🇺🇸

Buffalo, New York, United States

Medical University of South Carolina(MUSC)

🇺🇸

Charleston, South Carolina, United States

Wake Forest University- Forsyth Hospital and Brenner Hospital

🇺🇸

Winston-Salem, North Carolina, United States

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