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
Status: Recruiting
Location: See all (8) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Maximum Age: 10 days
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
Belgium
Cliniques Universitaires Saint-Luc (UCLouvain)
NOT_YET_RECRUITING
Brussels
ISPPC CHU Charleroi
NOT_YET_RECRUITING
Charleroi
Clinique CHC Montlégia
NOT_YET_RECRUITING
Liège
Italy
AOU Careggi
ACTIVE_NOT_RECRUITING
Florence
IRCCS Instituto Giannina Gaslini
RECRUITING
Genova
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
ACTIVE_NOT_RECRUITING
Milan
AOU Policlinico di Modena
NOT_YET_RECRUITING
Modena
Unità di Fase I della UOC Terapia Intensiva e Patologia Neonatale, Assistenza Neonatale (TINI) dell'Azienda Ospedale Università di Padova
RECRUITING
Padua
Contact Information
Primary
Beatrice De Vos, M.D., Ph.D.
b.devos@exobio.be
+32 478 88 26 57
Backup
Lorine Preud'homme, M.Sc.
l.preudhomme@exobio.be
+32 468 07 40 71
Time Frame
Start Date:2023-12-28
Estimated Completion Date:2029-12-31
Participants
Target number of participants:265
Treatments
Experimental: EXOB-001 (Phase 1)
EXOB-001 will be administered via the endotracheal route in an already intubated newborn. EXOB-001 will be administered in three dose levels (low dose, medium dose and high dose) with one or three administrations.
Experimental: Active group 1 EXOB-001 (Phase 2)
In phase 2, active group 1 consists of administering the first (out of two) of the safest selected dose/regimen of EXOB-001 based on phase 1 interim results.
Experimental: Active group 2 EXOB-001 (Phase 2)
In phase 2, active group 2 consists of administering the second (out of two) of the safest selected dose/regimen of EXOB-001 based on phase 1 interim results.
Placebo_comparator: Placebo (Phase 2)
In phase 2, the saline solution for infusion is used as a placebo.