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Recruiting Phase 1/2

Safety and Efficacy of MSC-EVs in the Prevention of BPD in Extremely Preterm Infants

NCT06279741 · tracked via the Priya Life Science Italy tracker
Sponsor
EXO Biologics S.A
Phase
Phase 1/2
Started
2023-12-28
Last updated
2024-09-19

Condition(s) studied

Bronchopulmonary Dysplasia

Investigational drug(s) / intervention(s)

Endotracheopulmonary Instillation, Suspension

Endotracheopulmonary Instillation, Suspension: The endotracheal administration can be either by endotracheal tube instillation using a 5 French end-hole catheter or by endotracheal tube instillation using the secondary lumen of a dual lumen endotracheal tube. The dose is adjusted to body weight and the endotracheal administration will be performed in an already intubated newborn infant.

Study summary

The phase 1/2 trial aims to evaluate the safety and efficacy of EXOB-001 consisting of extracellular vesicles derived from umbilical cord mesenchymal stromal cells in the prevention of bronchopulmonary dysplasia (BPD) in extremely premature neonates. The study population includes babies born between 23 and 28 (27 + 6 days) weeks of gestational age and body weight between 500g and 1,500 g. Thirty-six subjects will receive one or three administrations of the three doses of EXOB-001 via the endotracheal route in phase 1. In phase 2, two dosages based on the results of phase 1 will be selected and a total of 203 subjects will be randomised to receive either EXOB-001 or placebo (saline solution).

Infants will be followed up to 2 years of corrected age (end of study).

Eligibility

Sex
ALL
Min age
—
Max age
10 Days
Healthy volunteers
No
Inclusion Criteria: * 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, - Not expected to be extubated within the next 24/48 hours after enrolment. * Written informed consent from parents/legally designated representative. Exclusion Criteria: * Surfactant administration less than 24 hours prior to (first) IMP administration. * 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 (IVH). * Has active pulmonary haemorrhage. * Has periventricular leukomalacia (PVL). * 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.

Primary outcome measure(s)

  • Number of subjects with treatment-emergent adverse events (phase 1) — From EXOB-001 administration up to 36 weeks post-menstrual age (PMA)
    The proportion of subjects exhibiting acute and short-term safety of the intratracheal administration of EXOB-001 (single dose or multiple doses at different dose levels).
  • Number of subjects with BPD grade II-III incidence rate per groups (phase 2). — 36 weeks PMA
    BPD grade II-III incidence rate per group assessed at 36 weeks PMA. The severity of BPD is assessed according to the modified NICHD severity grading (Grade I to IIIA) definition.

Trial sites (8)

FacilityCityRegionStatus
Cliniques Universitaires Saint-Luc (UCLouvain) Brussels Belgium Not Yet Recruiting
ISPPC CHU Charleroi Charleroi Belgium Not Yet Recruiting
Clinique CHC Montlégia Liège Belgium Not Yet Recruiting
AOU Careggi Florence Italy Active Not Recruiting
IRCCS Instituto Giannina Gaslini Genova Italy Recruiting
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy Active Not Recruiting
AOU Policlinico di Modena Modena Italy Not Yet Recruiting
Unità di Fase I della UOC Terapia Intensiva e Patologia Neonatale, Assistenza Neonatale (TINI) dell'Azienda Ospedale Università di Padova Padua Italy Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06279741 on ClinicalTrials.gov ↗ ← All trials in Italy