A Randomized, Controlled, Blinded, Parallel Group Study of the Safety and Efficacy of APC-0101 (SF-RI 1 Surfactant for Inhalation Combined With a Dedicated Delivery System) in Preterm Infants With Respiratory Distress Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 520
- 试验地点
- 12
- 主要终点
- Failure Criteria
研究概览
简要总结
This is a 2-part, prospective, randomized, blinded, sham-controlled, multi-center study comparing preterm subjects with RDS who are treated with APC-0101 and nCPAP/NIV to subjects treated with nCPAP/NIV alone (Sham). In Part 1, subjects will be followed until they reach 40 weeks post-menstrual age (PMA) or are discharged from the NICU, whichever comes first. In Part 2, subjects will undergo post-term follow-up through 24 months corrected age.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Blinding/masking will be accomplished by a two-level redundant process. First, a screen (or private room) will be placed around the subject's bedside area and the APC-0101 Controller during APC-0101 or Sham treatment. This will ensure that no members of the clinical team, other than the blinded bedside nurse, will see the subject or the Controller throughout treatment. Second, the Controller and subject's face will be shielded so the blinded bedside nurse cannot determine whether the subject is receiving APC-0101 or Sham treatment.
A trained, unblinded, staff member (e.g., a respiratory therapist) will set up the Controller, but will not be involved with making decisions about the respiratory support of the subject.
Parents and all members of the clinical team who will make decisions about respiratory support, including FiO2, assessment of Failure Criteria, or assessment of AEs, will remain blinded throughout the study.
入排标准
- 年龄范围
- 1 Hour 至 24 Hours（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Inborn at the study site's hospital (i.e., not transferred from another hospital following delivery)
- •Gestational age at birth of 26 through 33 weeks PMA
- •Birth weight appropriate for gestational age (AGA, weight 3rd to 97th percentile on Fenton Growth Curve)
- •Birth weight ≤ 2000 grams
- •Post-natal age 1 to 24 hours at randomization
- •On nCPAP or NIV for at least 30 minutes with RSS = 1.4 - 2.0 to maintain SpO2 90-95% at randomization. RSS is calculated as (nCPAP cm H2O) × (FiO2) or as (NIV mean airway pressure cm H2O) × (FiO2).
- •FiO2 ≥ 0.24 at randomization
- •nCPAP or mPaw ≥ 6 cm H2O at randomization
- •Chest radiograph (CXR) or lung ultrasound compatible with RDS prior to randomization
排除标准
- •On SiPAP®, RAM® cannula, or high flow nasal cannula (HFNC) (> 2 liters per minute [LPM]) at the time of randomization
- •Prior instillation of surfactant
- •Premature rupture of membranes (PROM) occurring > 14 days before birth
- •Significant congenital/chromosomal anomaly (e.g., Pierre Robin syndrome, clinically significant congenital heart disease, or trisomy)
- •Pneumothorax
- •Other etiologies of respiratory distress
- •Enrollment in another interventional study with similar efficacy endpoints
- •Apgar score at 5 min of 0-3
- •Prior cardiopulmonary resuscitation (CPR) or epinephrine
- •Base Deficit > 15 mEq/L on most recent arterial blood gas (not capillary blood gas, venous blood gas, or cord gas) prior to randomization. Note that arterial blood gas is not required prior to randomization.
- •Partial pressure of carbon dioxide (PaCO2) > 65 mmHg on most recent arterial blood gas (not capillary blood gas, venous blood gas, or cord gas) prior to randomization.
- •Triplet or higher order multiple birth
研究组 & 干预措施
Control Group
干预措施: Control (Other)
APC-0101 Group
干预措施: APC-0101 (SF-RI 1 surfactant for inhalation combined with a dedicated delivery system) (Combination Product)
结局指标
主要结局
Failure Criteria
时间窗: First 7 days of life
Number (%) of subjects who meet the predetermined criteria for failure of nCPAP/NIV
次要结局
- Instilled Bolus Surfactant(First 72 hours of life)
- Multiple Bolus Surfactant Doses(First 72 hours of life)
- Total Number of Bolus Surfactant Doses(First 72 hours of life)