protocolSection identificationModule nctId: NCT06279741, orgStudyIdInfo id: EVENEW, secondaryIdInfos id: 2022-500293-34, type: EUDRACT_NUMBER, secondaryIdInfos id: U1111-1291-0283, type: OTHER, domain: Universal Trial Number, briefTitle: Safety and Efficacy of MSC-EVs in the Prevention of BPD in Extremely Preterm Infants, acronym: EVENEW, statusModule overallStatus: RECRUITING, startDateStruct date: 2023-12-28, primaryCompletionDateStruct date: 2027-01-31, completionDateStruct date: 2029-12-31, studyFirstPostDateStruct date: 2024-02-28, lastUpdatePostDateStruct date: 2024-02-28, sponsorCollaboratorsModule leadSponsor name: EXO Biologics S.A., class: INDUSTRY, descriptionModule briefSummary: 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)., conditionsModule conditions: Bronchopulmonary Dysplasia, designModule studyType: INTERVENTIONAL, phases: PHASE1, phases: PHASE2, designInfo allocation: RANDOMIZED, interventionModel: PARALLEL, interventionModelDescription: Phase 1 is an open-labelled, dose-escalated, and single-arm of EXOB-001. Phase 2 is a randomised, double-blind, placebo-controlled, and dose-finding of EXOB-001., primaryPurpose: PREVENTION, maskingInfo masking: DOUBLE, maskingDescription: Phase 1 is an open-label, dose-escalation and single-arm study. In phase 2, Investigators will remain blinded to each subject's assigned treatment throughout the study. The Sponsor will put in place procedures to maintain this blind. Indeed, to ensure the blinding of the groups, the preparation and administration of the test product will be organized by different teams. In the event of a Quality Assurance audit, the auditor(s) will be allowed access to unblinded study treatment records at the site(s) to verify that randomisation/dispensing has been done accurately. Blinding will be broken by the Investigator for emergency purposes only, where knowledge of the blinded treatment could influence further subject care. In addition, subjects will be unblinded for safety reports, as per regulatory requirements. Study blind will be broken after the database lock., whoMasked: PARTICIPANT, whoMasked: INVESTIGATOR, enrollmentInfo count: 265, type: ESTIMATED, armsInterventionsModule interventions name: Endotracheopulmonary Instillation, Suspension, outcomesModule primaryOutcomes measure: Number of subjects with treatment-emergent adverse events (phase 1), primaryOutcomes measure: Number of subjects with BPD grade II-III incidence rate per groups (phase 2)., secondaryOutcomes measure: Assessment of medium-term safety of EXOB-001 (phase 1/2), secondaryOutcomes measure: Number of subjects with dose-limiting toxicity (DLT) (phase 1), secondaryOutcomes measure: Number of subjects needing for oxygen and ventilation for BPD incidence (phase 1/2), secondaryOutcomes measure: Assessment of immune markers (phase 2), secondaryOutcomes measure: Assessment of BPD incidence and severity (phase 1/2), secondaryOutcomes measure: Safety evaluation (phase 1/2), secondaryOutcomes measure: Assessment of lung ultrasound score (phase 1/2), secondaryOutcomes measure: Number of subjects with complications of prematurity (phase 1/2), secondaryOutcomes measure: Assessment of the respiratory morbidity (phase 1/2), secondaryOutcomes measure: Assessment of neurodevelopment (phase 1/2), eligibilityModule sex: ALL, maximumAge: 10 Days, stdAges: CHILD, contactsLocationsModule locations facility: Cliniques Universitaires Saint-Luc (UCLouvain), status: NOT_YET_RECRUITING, city: Brussels, zip: 1200, country: Belgium, contacts name: Olivier Danhaive, M.D., role: CONTACT, phone: +32 2 764 79 00, email: olivier.danhaive@saintluc.uclouvain.be, contacts name: Olivier Danhaive, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 50.85045, lon: 4.34878, locations facility: ISPPC CHU Charleroi, status: NOT_YET_RECRUITING, city: Charleroi, zip: 6000, country: Belgium, contacts name: Serge Vanden Eijnden, M.D., role: CONTACT, phone: +32 7 192 12 43, email: serge.vandeneijnden@chu-charleroi.be, contacts name: Serge Vanden Eijnden, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 50.41136, lon: 4.44448, locations facility: Clinique CHC Montlégia, status: NOT_YET_RECRUITING, city: Liège, zip: 4000, country: Belgium, contacts name: Pierre Maton, M.D., role: CONTACT, phone: +32 4 355 56 17, email: pierre.maton@chc.be, contacts name: Pierre Maton, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 50.63373, lon: 5.56749, locations facility: AOU Careggi, status: NOT_YET_RECRUITING, city: Florence, country: Italy, contacts name: Carlo Dani, M.D., role: CONTACT, phone: +39 055 7948421, email: carlo.dani@unifi.it, contacts name: Carlo Dani, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 43.77925, lon: 11.24626, locations facility: IRCCS Instituto Giannina Gaslini, status: NOT_YET_RECRUITING, city: Genova, country: Italy, contacts name: Giorgia Brigati, M.D., role: CONTACT, phone: +39 010 56362218, email: giorgiabrigati@gaslini.org, contacts name: Giorgia Brigati, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 44.40478, lon: 8.94438, locations facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, status: NOT_YET_RECRUITING, city: Milan, country: Italy, contacts name: Anna Lavizzari, M.D., role: CONTACT, email: anna.lavizzari@policlinico.mi.it, contacts role: CONTACT, phone: +39 025 5032907, contacts name: Anna Lavizzari, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 45.46427, lon: 9.18951, locations facility: AOU Policlinico di Modena, status: NOT_YET_RECRUITING, city: Modena, country: Italy, contacts name: Alberto Berardi, M.D., role: CONTACT, phone: +39 333 1053301, email: alberto.berardi@unimore.it, contacts name: Alberto Berardi, M.D., role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 44.64783, lon: 10.92539, locations facility: Unità di Fase I della UOC Terapia Intensiva e Patologia Neonatale, Assistenza Neonatale (TINI) dell'Azienda Ospedale Università di Padova, status: RECRUITING, city: Padua, zip: 35128, country: Italy, contacts name: Eugenio Baraldi, Prof, role: CONTACT, phone: +39 049 8213578, email: eugenio.baraldi@unipd.it, contacts name: Eugenio Baraldi, Prof, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 45.40797, lon: 11.88586, hasResults: False