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Allogeneic Umbilical Cord Mesenchymal Cells-Derived Extracellular Vesicles

This article discusses an innovative clinical trial investigating the use of Allogeneic Umbilical Cord Mesenchymal Cells-Derived Extracellular Vesicles (EXOB-001) in preventing bronchopulmonary dysplasia (BPD) in extremely preterm newborns. The study aims to assess the safety and efficacy of this novel treatment approach, which could potentially improve outcomes for premature infants at risk of developing lung complications.

Table of Contents

What is EXOB-001?

EXOB-001 is a new medical treatment being studied for extremely preterm newborns. It’s made from special particles called extracellular vesicles that come from umbilical cord mesenchymal cells[1]. These cells are collected from donated umbilical cords, so the treatment is considered “allogeneic,” which means it comes from a donor rather than the patient themselves.

The full name of this treatment is “Allogeneic Umbilical Cord Mesenchymal Cells-Derived Extracellular Vesicles,” but it’s easier to remember it as EXOB-001. It’s also sometimes called by its sponsor product code, which is the same: EXOB-001[1].

What is Bronchopulmonary Dysplasia (BPD)?

Bronchopulmonary dysplasia (BPD) is a serious lung condition that affects some premature babies, especially those born extremely early (before 28 weeks of pregnancy)[1]. When babies are born this early, their lungs are not fully developed. They often need help breathing with machines and extra oxygen. While this support is necessary, it can sometimes damage their delicate lungs, leading to BPD.

Babies with BPD may have trouble breathing and need oxygen support for a long time. This can affect their growth and development, and may lead to long-term health problems[1].

How EXOB-001 Works

EXOB-001 is given to babies through their breathing tube directly into their lungs. This method is called intratracheal administration[1]. The extracellular vesicles in EXOB-001 are tiny particles that can carry important signals and materials between cells. Scientists believe these vesicles might help reduce inflammation and promote healing in the lungs of premature babies, potentially preventing or reducing the severity of BPD.

Clinical Trial Overview

EXOB-001 is currently being studied in a clinical trial called EVENEW. This is a “first in human” trial, which means it’s the first time this treatment is being tested in people[1]. The trial has two main parts:

  1. Phase I: This part focuses on testing the safety of EXOB-001 and finding the right dose. Researchers will carefully monitor babies for any side effects[1].
  2. Phase II: This part will compare EXOB-001 to a placebo (a harmless substance with no active ingredients) to see if it helps prevent or reduce BPD[1].

Who Can Participate in the Trial?

The trial is looking for extremely premature babies who meet specific criteria. Some key points include:

  • Babies must be between 23 and 28 weeks gestational age at birth
  • They must weigh between 500g and 1500g at birth
  • They need to be on a breathing machine with extra oxygen
  • They must be between birth and 10 days old when starting the trial[1]

There are also several reasons why a baby might not be able to participate, such as having certain heart defects, lung malformations, or infections[1].

Potential Benefits of EXOB-001

While it’s important to remember that EXOB-001 is still being studied and its benefits are not yet proven, researchers hope it might:

  • Reduce the number of babies who develop severe BPD
  • Help babies need less time on breathing machines
  • Improve lung development
  • Reduce the risk of other complications related to prematurity[1]

Safety Considerations

The main goal of the Phase I part of the trial is to check the safety of EXOB-001. Researchers will be watching closely for any side effects, especially in the first few hours and days after the treatment is given. They’ll be looking at things like changes in heart rate, blood pressure, and oxygen levels[1].

Long-term Follow-up

The trial plans to follow the babies for up to 2 years after they would have been born if they hadn’t been premature (called “corrected age”). During this time, researchers will check on the babies’ overall health, especially their lung health. They’ll also look at the babies’ development using special questionnaires for parents[1].

This long-term follow-up is important to understand not just the immediate effects of EXOB-001, but also how it might impact children’s health and development as they grow.

Aspect Details
Study Type Phase I single arm, dose escalating and Phase II double blind, randomized, placebo-controlled dose finding clinical trial
Intervention Intratracheal administration of EXOB-001 (Allogeneic Umbilical Cord Mesenchymal Cells-Derived Extracellular Vesicles)
Target Condition Bronchopulmonary Dysplasia (BPD) in extremely preterm newborns
Primary Objectives Phase I: Assess acute and short-term safety of EXOB-001
Phase II: Assess efficacy in reducing BPD grade II-III at 36 weeks postmenstrual age
Key Eligibility Criteria – 23-28 weeks gestational age at birth
– Birth weight 500-1500g
– Requiring mechanical ventilation
Follow-up Duration Up to 2 years corrected age
Key Outcomes – Safety and tolerability of EXOB-001
– BPD incidence and severity
– Lung development and function
– Neurodevelopmental outcomes
– Respiratory morbidity

FAQ

  • What is EXOB-001 and how is it administered? EXOB-001 is a medication derived from extracellular vesicles of umbilical cord mesenchymal cells. It is administered through intratracheal instillation, which means it is delivered directly into the windpipe of the preterm newborn.
  • What is the main goal of this clinical trial? The main goal is to assess the safety and effectiveness of EXOB-001 in preventing bronchopulmonary dysplasia (BPD) in extremely preterm newborns. The trial aims to determine if this treatment can reduce the severity and incidence of BPD compared to a placebo.
  • Who is eligible to participate in this trial? Eligible participants are newborns up to 10 days old, born between 23 and 28 weeks of gestation, weighing between 500g and 1500g, who require mechanical ventilation and are not expected to be taken off the ventilator within the next 24-48 hours.
  • What are the potential benefits of this treatment? If successful, EXOB-001 could help prevent or reduce the severity of bronchopulmonary dysplasia in extremely preterm newborns. This may lead to improved lung development, shorter hospital stays, and better long-term health outcomes for these vulnerable infants.
  • How long will the participants be monitored in this study? Participants will be monitored closely during their hospital stay and followed up for up to 2 years corrected age. This long-term follow-up will assess overall health status, with special attention to pulmonary morbidity, neurodevelopmental condition, and respiratory symptoms.

Ongoing Clinical Trials on Allogeneic Umbilical Cord Mesenchymal Cells-Derived Extracellular Vesicles

  • Study on the Safety and Effect of EXOB-001 in Preventing Bronchopulmonary Dysplasia in Extremely Preterm Newborns

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Italy

Glossary

  • Bronchopulmonary Dysplasia (BPD): A chronic lung disease that primarily affects premature infants who require mechanical ventilation or oxygen therapy. It is characterized by inflammation and scarring in the lungs, which can lead to long-term breathing difficulties.
  • Extracellular Vesicles: Tiny particles released by cells that can carry various molecules, including proteins and genetic material. In this study, they are derived from umbilical cord mesenchymal cells and are being investigated for their potential therapeutic effects.
  • Intratracheal Administration: A method of delivering medication directly into the windpipe (trachea). In this study, EXOB-001 is administered using this technique to target the lungs of preterm newborns.
  • Gestational Age: The length of pregnancy from the first day of the last menstrual period to birth. In this study, eligible infants are born between 23 and 28 weeks of gestation, which is considered extremely preterm.
  • Mechanical Ventilation: A life support treatment that helps a patient breathe using a machine called a ventilator. It is often necessary for extremely preterm infants whose lungs are not fully developed.
  • Placebo: An inactive substance used in clinical trials to compare the effects of a new treatment. In this study, some infants will receive a saline solution as a placebo instead of EXOB-001.
  • Postmenstrual Age (PMA): A measure of age used for preterm infants, calculated by adding the gestational age at birth to the time elapsed since birth. In this study, some assessments are made at 36 weeks PMA.
  • Respiratory Severity Score (RSS): A measure used to assess the severity of respiratory problems in newborns. It takes into account factors such as oxygen requirements and ventilator settings.
  • Neurodevelopmental Condition: The status of a child's brain and nervous system development, including cognitive, motor, and social skills. This study will evaluate neurodevelopment up to 2 years corrected age.
  • Corrected Age: The age of a preterm infant calculated from their due date rather than their actual birth date. It is used to more accurately assess development in premature infants.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effect-of-exob-001-in-preventing-bronchopulmonary-dysplasia-in-extremely-preterm-newborns/