A Multicenter, Randomized, Open-label, Controlled Trial To Assess The Safety And Tolerability Of Lucinactant For Inhalation In Preterm Neonates 26 to 28 Weeks PMA
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 48
- 试验地点
- 40
- 主要终点
- Number of Participants With Peri-Dosing Adverse Events - Initial Dose
研究概览
简要总结
This study is to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in up to four escalating doses to preterm neonates 26 to 28 weeks gestational age who are receiving nCPAP for RDS compared to neonates receiving nCPAP alone.
详细描述
This study was a multicenter, randomized, controlled, open-label, dose-escalation study, conducted to evaluate the safety and tolerability of lucinactant for inhalation in conjunction with nasal continuous positive airway pressure (nCPAP) in comparison with nCPAP alone. The study was to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in 4 escalating doses.
For this study, lucinactant for inhalation refers to the active investigational agent, lyophilized lucinactant, in combination with the prototype investigational delivery device. Reconstituted lyophilized lucinactant was aerosolized by the investigational device and introduced into the nCPAP circuit. Those randomized to the control arm continued to receive nCPAP alone. Dose assignments were unblinded, as the primary objective of this study was safety and tolerability.
Preterm neonates with respiratory distress syndrome (RDS) between 26 and 28 completed weeks PMA who were within the first 20 hours after birth and who had successful implementation of controlled nCPAP within 90 minutes of birth were considered to be potential subjects. Before study enrollment, legal guardians were provided a written informed consent form (ICF) for each potential subject.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 26 Weeks 至 28 Weeks（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent from a legally authorized representative.
- •Gestational age 26 to 28 completed weeks post menstrual age (PMA).
- •Successful implementation of controlled nCPAP within 90 minutes after birth.
- •Spontaneous breathing.
- •Chest radiograph consistent with RDS.
- •Within the first 20 hours after birth, have an nCPAP of 5 to 6 cm H2O to maintain oxygen saturation measured by pulse oximetry (SpO2) of 88% to 95% with a fraction of inspired oxygen (FiO2) of 0.25 to 0.50 that is clinically indicated for at least 30 minutes. Transient (<10 minutes) FiO2 excursions below 0.25 or above 0.50 did not reset the 30 minute requirement.
排除标准
- •Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth.
- •Recurrent episodes of apnea occurring after the initial newborn resuscitation period (ie, 10 minutes after birth) requiring intermittent positive pressure breaths using inflating pressures above the set CPAP pressure administered manually or mechanically through any patient interface.
- •A 5 minute Apgar score <
- •Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth.
- •Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH).
- •Known or suspected chromosomal abnormality or syndrome.
- •Premature rupture of membranes (PROM) > 2 weeks.
- •Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.
- •Need for endotracheal intubation and mechanical ventilation.
- •Has been administered: another investigational agent or investigational medical device, any other surfactant agent, steroid treatment after birth.
研究组 & 干预措施
nCPAP alone
nCPAP therapy alone
干预措施: nCPAP alone (Device)
150 mg/kg
Lucinactant for inhalation 150 mg TPL/kg with nCPAP
1 repeat dose will be allowed if repeat dosing criteria are met.
干预措施: Lucinactant for inhalation (Combination Product)
50 mg/kg
Lucinactant for inhalation 50 mg total phospholipids (TPL)/kg with nCPAP
1 repeat dose allowed if repeat dosing criteria are met.
干预措施: Lucinactant for inhalation (Combination Product)
75 mg/kg
Lucinactant for inhalation 75 mg TPL/kg with nCPAP
1 repeat dose allowed if repeat dosing criteria are met.
干预措施: Lucinactant for inhalation (Combination Product)
100 mg/kg
Lucinactant for inhalation 100 mg TPL/kg with nCPAP
1 repeat dose allowed if repeat dosing criteria are met.
干预措施: Lucinactant for inhalation (Combination Product)
结局指标
主要结局
Number of Participants With Peri-Dosing Adverse Events - Initial Dose
时间窗: Randomization to 24 Hours Post Randomization
Number of Participants with adverse events that were experienced during the initial study treatment
Number of Participants With Air Leak
时间窗: 7 days
Number of participants with air leak (includes pneumothorax, pulmonary interstitial emphysema (PIE), pneumomediastinum, pneumopericardium, subcutaneous emphysema)
次要结局
- Number of Participants With Complications of Prematurity(Randomization to 36 weeks PMA)
- Number of Participants With Worsening of Respiratory Status Criteria(Randomization to 72 Hours Post Randomization)
- Bronchopulmonary Dysplasia(Randomization to 36 weeks PMA)
- Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure(Randomization to 72 Hours Post Randomization)
- Death(Randomization to 36 weeks PMA)
- FiO2(Randomization to 72 hours post randomization)