Using extracellular vesicles to prevent lung disease in premature infants
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 Cord Mesenchymal Cells-derived Extracellular Vesicles in Preventing Bronchopulmonary Dysplasia in Extremely Preterm Newborns
This study is testing a new treatment using special cells from umbilical cords to see if it can help prevent lung disease in very premature infants.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 265 (estimated) |
| Ages | N/A to 10 Days |
| Sex | All |
| Sponsor | EXO Biologics S.A. Industry-sponsored |
| Locations | 8 sites (Brussels and 7 other locations) |
| Trial ID | NCT06279741 on ClinicalTrials.gov |
What this trial studies
This phase 1/2 trial evaluates the safety and efficacy of EXOB-001, which consists of extracellular vesicles derived from umbilical cord mesenchymal stromal cells, in preventing bronchopulmonary dysplasia (BPD) in extremely premature infants. The study will involve infants born between 23 and 28 weeks of gestational age and weighing between 500g and 1,500g. Participants will receive one to three doses of EXOB-001 via endotracheal administration, with follow-up extending to two years of corrected age. The trial aims to determine the optimal dosage based on initial safety and efficacy results.
Who should consider this trial
Good fit: Ideal candidates are extremely preterm infants aged 0 to 10 days, born between 23 and 28 weeks of gestation, and requiring mechanical ventilation.
Not a fit: Patients with congenital heart defects or serious lung malformations may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the incidence of bronchopulmonary dysplasia in extremely preterm infants, improving their long-term respiratory health.
How similar studies have performed: While the use of extracellular vesicles in this context is relatively novel, preliminary evidence suggests potential efficacy in similar approaches.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Brussels and 7 other locations
- 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)
Study contacts
- Study coordinator: Beatrice De Vos, M.D., Ph.D.
- Email: b.devos@exobio.be
- Phone: +32 478 88 26 57
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.