Ignite Creation Date:
2024-05-06 @ 8:10 PM
Last Modification Date:
2024-10-26 @ 3:22 PM
Study NCT ID:
NCT06279741
Status:
RECRUITING
Last Update Posted:
2024-02-28
First Post:
2024-02-01
Brief Title:
Safety and Efficacy of MSC-EVs in the Prevention of BPD in Extremely Preterm Infants
Sponsor:
EXO Biologics SA
Organization:
EXO Biologics SA
Study Overview
Official Title:
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
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
EVENEW
Brief Summary:
The phase 12 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 1500 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
Infants will be followed up to 2 years of corrected age end of study
Detailed Description:
Bronchopulmonary Dysplasia BPD is a chronic severe multifactorial respiratory disease that affects extremely premature infants and is the most common and severe consequence of preterm birth BPD is associated with disrupted alveolarization and microvascular development resulting in abnormal gas exchange and lung mechanics BPD has a multifactorial aetiology with pre- peri- and postnatal mechanisms causing inflammation and injury and resulting in the disruption of the lungs development with the insurgence of an aberrant repair mechanism
EXOB-001 consists of a population of EVs smaller than 022 μm in diameter containing proteins and nucleic acids enclosed in a double layer of phospholipids with integral and surface-bound proteins as the main components EVs exert anti-inflammatory and immunomodulatory activity by reducing the release of proinflammatory cytokines and reducing the recruitment of immune cells in the lung Current evidence shows that EVs can modulate macrophage phenotype and this is relevant for BPD because of the role macrophages have in its pathogenesis
Two hundred sixty-five 265 40 in phase 1 to reach 36 evaluable subjects 225 in phase 2 to reach 203 evaluable subjects extremely preterm infants at risk of developing BPD with 23 weeks up to 28 27 weeks6 days weeks of gestational age and birth weight between 500g and 1500g and being endotracheally intubated between postnatal day 3 and day 10 receiving mechanical ventilation with FiO2 25
Phase 1 will start with cohorts with a single administration starting with a low dose up to a high dose and thereafter start the escalation of cohorts with 3 administrations starting with a low dose up to a high dose In the case of 3 endotracheal administrations there will be a window of 24 hours between the administrations the maximal duration of the treatment with EXOB-001 will be 48 hours
Phase 2 includes 2 groups with selected dosage levels and regimen of EXOB-001 based on phase 1 interim results Subjects will be randomised 221 to receive either EXOB-001 or placebo saline solution
EXOB-001 consists of a population of EVs smaller than 022 μm in diameter containing proteins and nucleic acids enclosed in a double layer of phospholipids with integral and surface-bound proteins as the main components EVs exert anti-inflammatory and immunomodulatory activity by reducing the release of proinflammatory cytokines and reducing the recruitment of immune cells in the lung Current evidence shows that EVs can modulate macrophage phenotype and this is relevant for BPD because of the role macrophages have in its pathogenesis
Two hundred sixty-five 265 40 in phase 1 to reach 36 evaluable subjects 225 in phase 2 to reach 203 evaluable subjects extremely preterm infants at risk of developing BPD with 23 weeks up to 28 27 weeks6 days weeks of gestational age and birth weight between 500g and 1500g and being endotracheally intubated between postnatal day 3 and day 10 receiving mechanical ventilation with FiO2 25
Phase 1 will start with cohorts with a single administration starting with a low dose up to a high dose and thereafter start the escalation of cohorts with 3 administrations starting with a low dose up to a high dose In the case of 3 endotracheal administrations there will be a window of 24 hours between the administrations the maximal duration of the treatment with EXOB-001 will be 48 hours
Phase 2 includes 2 groups with selected dosage levels and regimen of EXOB-001 based on phase 1 interim results Subjects will be randomised 221 to receive either EXOB-001 or placebo saline solution
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2022-500293-34 | EUDRACT_NUMBER | None | None |
| U1111-1291-0283 | OTHER | Universal Trial Number | None |