Bronchopulmonary Dysplasia (BPD) MedDRA version: 18.0 Level: PT Classification code 10006475 Term: Bronchopulmonary dysplasia System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Interventions
Product Name: rhCC10
Pharmaceutical Form: Endotracheopulmonary instillation, solution
INN or Proposed INN: Not assigned yet
Current Sponsor code: rhCC10
Concentration unit: mg/kg milligram(s)/kilogram
Sponsors
Therabron Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Age = 24 hours; •Birthweight between 600 and 1,250 grams; •Gestational age 24-29 weeks (not less than 24 weeks); at birth based on best estimate using obstetrical sonography (first or second trimester), solid dating criteria, or Ballard examination; •Birthweight appropriate for gestational age; •5 minute Apgar score >5; •Diagnosis of neonatal RDS based on clinical and radiographic criteria; •Requiring intubation and mechanical ventilation for treatment of RDS; •Received at least one dose of surfactant (prophylaxis or rescue) or the decision to treat with surfactant has already been made (e.g. infant is intubated or in the process of being intubated and/or surfactant has been prescribed); and •Written informed consent is obtained from the infant’s parent or legal guardian prior to enrollment of the patient and agrees to all study-related procedures and evaluations, including those required after hospital discharge. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •5 minute Apgar score of = 5; except in cases where the subject’s Apgar score is = 5 at 5 minutes, normalizes by 10 minutes, no acidosis is present on cord blood gas and the neonatal depression is thought to be due to maternal anesthesia or other drug exposure such as MgSO4. •Major congenital abnormalities (chromosomal, renal, cardiac, hepatic, neurologic, or pulmonary malformations; minor anomalies such as cleft lip/palate are permitted); •Evidence of severe neonatal depression (as defined by cord blood pH = 7.00 and/or an Apgar score of < 4 at 10 minutes); •Evidence of congenital infection (bacterial or non-bacterial) ; •Requires a major surgical procedure prior to administration of Study drug •Enrollment in any other study involving administration of another investigational drug; •Any condition which could preclude receiving study drug or performing any study-related procedures; •Use of postnatal corticosteroids prior to administration of rhCC10, except as specified in the protocol; •Use of inhaled nitric oxide prior to administration of rhCC10; •Mother is known to be seropositive for HIV or HTLV-1 (per maternal medical records); •Parent or guardian is unable or unwilling to complete the study diary after hospital discharge; •Parent or guardian is unable to bring the infant back to the study center for follow-up evaluations after discharge.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the efficacy of a single intratracheal dose of rhCC10 (1.5mg/kg and 5mg/kg in a 2 ml/kg fixed volume) in improving survival without chronic respiratory morbidity as indicated by a reduction in respiratory complications at 12 months corrected gestational age.;Secondary Objective: Not applicable;Primary end point(s): The primary outcome of the study will be survival without CRM at 12 months CGA as measured by a validated respiratory diary based scoring system (presence of wheezing and/or coughing 2 days per week for 3 consecutive weeks) and pulmonary questionnaires (decrease in respiratory illness requiring medications, unscheduled medical visits and/or ER or hospital admissions) which have been shown to correlate closely with abnormalities on pulmonary function testing. The 12 month CGA endpoint has been shown to be more predictive of respiratory morbidity at two years of life than a diagnosis of BPD at 36 weeks CGA in previous studies of high frequency oscillatory ventilation in preterm infants. ;Timepoint(s) of evaluation of this end point: 2 days per week for 3 consecutive weeks then once a month during 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1) Long Term Efficacy – 6 & 18 months A secondary outcome of the study will be survival without CRM at 6 months CGA as measured by validated respiratory diaries (presence of wheezing and/or coughing 2 days per week for 3 consecutive weeks) and pulmonary questionnaires (decrease in respiratory illness requiring medications and/or hospitalization) which have been shown to correlate closely with abnormalities on pulmonary function testing. Infants will be also be evaluated by physical examinations at 18 months CGA to evaluate growth and neurological development. 2) Short Term Efficacy Short term efficacy evaluations will include time of mechanical ventilation, oxygen requirement at 36 weeks CGA, and survival without BPD at 36 weeks CGA (or NICU discharge) as measured by oxygen challenge testing. Oxygen challenge testing involves the withdrawal of supplemental oxygen for a 30 minute period, during which the infant is closely monitored. At the end of the 30’ period, oxygenation is measured by pulse oximetry and the infant is placed back on supplemental oxygen. The infant’s BPD status is then determined by oxygen saturation; a diagnosis of BPD will be made if the infant’s oxygen saturation is 90% or less (<90%) at or before the end of the thirty minute period. If the infant’s oxygen level remains above 90% for the entire period, then the diagnosis will be “No BPD”. Failure to complete the test due to respiratory distress would be scored as severe BPD. The severity of BPD will be assessed using NIH consensus guidelines for infants born at <32 weeks PMA based on oxygen requirements at 28 days post-natal age and at 36 weeks PMA or at NICU discharge (whichever comes first). 3) Safety and Efficacy - Adverse Events The safety of the study drug will continue to be assessed by comparing the incidence of adverse events and serious adverse events in the treatment and placebo groups to each other and to the historical incidence of the adverse events | — |
Countries
France, Germany, Poland, United States
Contacts
Public ContactRené Mignolet
RLM Consulting
Outcome results
None listed