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临床试验/2022-500293-34-01
2022-500293-34-01招募中2 期

Phase I single arm, dose escalating and phase II double blind, randomized, placebo-controlled dose finding clinical trial assessing safety, and efficacy of intratracheal administration of allogeneic umbilical mesenchymal cells-derived extracellular vesicles in preventing bronchopulmonary dysplasia in extremely preterm newborns.

Exo Biologics11 个研究点 分布在 2 个国家目标入组 265 人开始时间: 2023年7月11日最近更新:
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适应症

试验速览

阶段
2 期
状态
招募中
发起方
Exo Biologics
入组人数
265
试验地点
11
主要终点
Phase I: Treatment-emergent adverse events (TEAEs) assessed from EXOB-001 administration up to 36 weeks PMA.
研究概览入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

Phase I primary objective To assess the acute and short-term safety of the intratracheal (IT) administration of EXOB-001 (single dose or multiple doses at different dose levels) at 36 weeks postmenstrual age (PMA).

Phase II primary objective To assess the efficacy of EXOB-001 on the reduction of BPD grade II-III (modified NICHD severity grading case definition) at 36 weeks PMA as compared to a placebo group (saline solution).

入排标准

年龄范围
0 years 至 17 years（0-17 Years）
接受健康志愿者
是

入选标准

  • •From birth up to 10 days chronological age
  • •From 23 weeks up to 28 weeks (27 week+6 days) gestational age at birth
  • •Birth weight ≥ 500g but ≤1500g
  • •Endotracheally intubated and receiving mechanical ventilation with FiO2 > 25% anytime between 3 and 10 days postnatally or needing re-intubation due to respiratory complications, and
  • •Not expected to be extubated within the next 24/48 hours after enrolment
  • •Written informed consent from parents/legally designated representative

排除标准

  • •Surfactant administration less than 24 hours prior to (first) IMP administration
  • •Has active pulmonary haemorrhage
  • •Has periventricular leukomalacia
  • •The subject is currently participating in any other interventional clinical study
  • •The subject is, in the opinion of the Investigator, so ill that death is inevitable, or is considered inappropriate for the study such as an infant that received thoracic compressions and/or adrenaline administration during stabilization in the delivery room and for any reason(s) other than those listed above
  • •Has a congenital heart defect, except for patent ductus arteriosus (PDA), atrial septal defect or a small/moderate, restrictive ventricular septal defect
  • •Has a serious malformation of the lung, such as pulmonary hypoplasia/aplasia, congenital diaphragmatic hernia, or any other congenital lung anomaly
  • •Being treated with inhaled nitric oxide
  • •Has a known chromosomal abnormality (e.g., Trisomy 18, Trisomy 13, or Trisomy 21) or a severe congenital malformation (e.g., hydrocephalus and encephalocele, trachea-oesophageal fistula, abdominal wall defects, and major renal anomalies)
  • •Has had a known severe congenital infectious disease (i.e., herpes, toxoplasmosis rubella, syphilis, human immunodeficiency virus, cytomegalovirus, etc.)
  • •Active systemic infection, severe sepsis, or septic shock at screening up to baseline (phase I) or randomization (phase II).
  • •Underwent a surgical procedure (requiring admission to an operating room) within 72 hours before baseline (phase I)/randomization (phase II) or who is anticipated to have a surgical procedure (requiring admission to an operating room) within 72 hours before or following baseline (phase I)/randomization (phase II)
  • •Has had a Grade 3 or 4 intraventricular haemorrhage

结局指标

主要结局

Phase I: Treatment-emergent adverse events (TEAEs) assessed from EXOB-001 administration up to 36 weeks PMA.

Phase I: Treatment-emergent adverse events (TEAEs) assessed from EXOB-001 administration up to 36 weeks PMA.

Phase II: BPD grade II-III incidence rate per groups assessed at 36 weeks PMA. Severity of BPD assessed according to the modified NICHD severity grading (Grade I to IIIA) definition.

Phase II: BPD grade II-III incidence rate per groups assessed at 36 weeks PMA. Severity of BPD assessed according to the modified NICHD severity grading (Grade I to IIIA) definition.

次要结局

  • Phase I: Treatment-emergent adverse events (TEAEs), and clinical examination and blood tests.
  • Phase I: Dose limiting toxicity measured as: cardiorespiratory decompensation (hypotension with tachycardia, lactic acidosis, oliguria, with echocardiographic confirmation if possible); decrease in SpO2/FiO2 ratio of more than 100 points or increase in FiO2 by > 30% (defined as 30% increase from the baseline, e.g. from 40% to 70%) that persist for at least 4 hours; stable (> 3 hours) increase in required mean airway pressure >3 cm H2O; death; anaphylactic reaction; any serious adverse reaction
  • Phase II: All adverse events (AEs) and serious adverse events (SAEs) (including case fatalities), both related and non-related.
  • Phase I: Need for oxygen and ventilation support (case definition according to Jobe and Bancalari 2001, case definition according to Isayama et al. 2017].
  • Phase I: The number of cases and severity of BPD measured according to modified NICHD severity grading (Grade I to IIIA) case definition and according to Jobe and Bancalari 2001 (mild, moderate, severe) case definition. The number of cases of BPD according to Isayama et al. 2017 case definition.
  • Phase I: Lung ultrasound (LUS).
  • Phase I: Chest X-Ray(s).
  • Phase I: Functional and anatomic echocardiography.
  • Phase I: Clinical examination, blood tests.
  • Phase I: Number of deaths caused by lung failure.
  • Phase I: All SAEs (including all-cause deaths).
  • Phase I: Duration of MV/respiratory support (i.e., total time receiving supplemental oxygen). Respiratory Severity Score. Duration of hospitalisation. Assessment of ROP, NEC, IVH, sepsis, PVL, HMD, apnoea, pneumothorax. Concomitant treatments, such as steroids, and surfactant. Concomitant procedures and therapies.
  • Phase I: Occurrence of ROP, NEC, IVH, sepsis, PVL, HMD, apnoea, pneumothorax; concomitant treatments such as steroids and others, procedures and therapies.
  • Phase I: ASQ3 Ages & Stages questionnaire for parents.
  • Phase I: Liverpool Respiratory Symptom Questionnaire (LRSQ).
  • Phase II: Need for oxygen and ventilation support, and BPD incidence (BPD case definition according to Isayama et al. 2017). Per group.
  • Phase II: Need for oxygen and ventilation support at day 28 chronological age, and BPD incidence and severity rate at 36 weeks PMA (BPD case definition according to Jobe and Bancalari 2001). Per group.
  • Phase II: BPD grade I-II-III incidence rate per group. Severity of BPD assessed according to the modified NICHD severity grading (Grade I to IIIA) definition.
  • Phase II: BPD grade II-III incidence rate in each EXOB-001 group. Severity of BPD assessed according to the modified NICHD severity grading (Grade I to IIIA) definition.
  • Phase II: Chest X-Ray(s). Per group.
  • Phase II: Functional and anatomic echocardiography. Per group.
  • Phase II: Clinical examination, blood tests. Per group.
  • Phase II: Lung ultrasound (LUS). Per group.
  • Phase II: Number of deaths caused by lung failure per group.
  • Phase II: All AEs and SAEs (related and non-related) per group.
  • Phase II: All SAEs (including all-cause deaths). Per group.
  • Phase II: Duration of MV/respiratory support and hospitalisation. Assessment of RSS. Assessment of ROP, NEC, IVH, sepsis, PVL, HMD, apnoea, pneumothorax. Concomitant treatments, such as steroids, and surfactant. Per group.
  • Phase II: Occurrence of ROP, NEC, IVH, sepsis, PVL, HMD, apnoea, pneumothorax. Concomitant treatments such as steroids and others, procedures, and therapies. Per group.
  • Phase II: IL-6, IL-8, TNFa, TGFb1, IL1b and IL1ra. Subset of subjects in all groups.
  • Phase II: ASQ3 Ages and Stages questionnaire for parents. Per group.
  • Phase II: Liverpool Respiratory Symptom Questionnaire (LRSQ). Per group.

研究者

发起方
Exo Biologics
申办方类型
Pharmaceutical company
责任方
Principal Investigator
主要研究者

Clinical Department

Scientific

Exo Biologics

研究点 (11)

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