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NCT Number: NCT07058025

Mesenchymal Stromal Cell Therapy to Prevent Bronchopulmonary Dysplasia in Extreme Preterm Infants

This clinical trial aims to evaluate the safety and efficacy of mesenchymal stromal cell (MSC) therapy in extreme preterm infants to prevent bronchopulmonary dysplasia, the main respiratory complication of preterm birth.

Study participants will receive either multiple intravenous doses (total of 3 doses) of MSC derived from human donor umbilical cord tissue (intervention group) or no uc-MSC injection (control group) to confirm the safety of IV MSC in extreme preterm infants and evaluate the potential benefit of MSC therapy on their respiratory health as well as on other complications related to preterm birth.

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Key information

Conditions

Bronchopulmonary Dysplasia (BPD) Bronchopulmonary Dysplasia Congenital, Hereditary, and Neonatal Diseases and Abnormalities ELGAN (22-28SA) Female Urogenital Diseases and Pregnancy Complications Infant, Newborn, Diseases Infant, Premature, Diseases Lung Diseases Lung Injury Obstetric Labor Complications Obstetric Labor, Premature Pregnancy Complications Premature Birth Respiratory Tract Diseases Urogenital Diseases Ventilator-Induced Lung Injury

Age range

4 day–14 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Royal Alexandra Hospital/Stollery Children's Hospital, Edmonton, Alberta, Canada

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About this study

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Gestational age (GA) less than 28+0 weeks
  • Post-natal age between 4 and 14 days of life
  • Invasive ventilation with oxygen requirement:
  • On mechanical ventilation: intubated patient with any of the following ventilation modes: conventional, HFO or Jet ventilation:
  • With requirement of FiO2: FiO2 >= 30% and for at least 12 hours over 24 hours (i.e. flowsheets, FiO2 histogram)

Exclusion criteria

  • Congenital anomaly:
  • Genetic and chromosomal syndromes (e.g., Trisomy 13, Trisomy 18, Trisomy 21): either patient with high suspicion (antenatal findings, clinical features) or documented syndrome by genetic testing.
  • Major congenital anomalies including cardiac (i.e., congenital heart defects, NB. PDA is not considered an exclusion criterion), neurological (e.g., holoprosencephaly, anencephaly), gastrointestinal (e.g., gastroschisis, omphalocele), pulmonary (e.g., congenital diaphragmatic hernia) anomalies.
  • Inborn errors of metabolism.
  • Hemodynamic instability (shock):
  • Hemodynamic instability with impaired end-organ perfusion (metabolic acidosis with increased lactate and/or decreased urine output).
  • Requirements for fluid bolus, inotrope or vasopressor medication
  • Severe sepsis:
  • Signs of hemodynamic instability and requiring at least one fluid bolus.
  • And a positive blood or cerebrospinal fluid culture.
  • Pneumothorax: Pneumothorax with a chest tube in place
  • Severe pulmonary hemorrhage:
  • Active pulmonary hemorrhage (i.e., frank blood coming from the endotracheal tube.
  • And at least one of the following criteria: a)hemodynamic instability. b) blood product transfusion (packed red blood cells, platelets, fresh frozen plasma)
  • Extubation: If Extubation planned within the next 24 hours (post first uc-MSC administration/sham procedure).
  • Patient is not expected to survive:
  • Redirection of care.
  • Patient is moribund

Treatment and study plan

Human Allogenic Umbilical Cord Mesenchymal Stromal Cells

Biological

IV administration of uc-MSC every 7 days ± 1 day for 3 weeks. Randomized double blinded

Other names: UC-MSC, Umbilical Cord Mesenchymal Stromal Cells, Mesenchymal Stromal Cells

Sham procedure control

Other

Sham procedure (mimic IV catheter insertion adn cell product infusion behing a screen). Repeated weekly for 3 weeks

Primary outcomes

  1. Number of mechanical ventilation-free days accounting for mortality

    Time frame: 120 days

    The study primary outcome is the number of mechanical ventilation-free days accounting for mortality. Mechanical ventilation is defined by artificial ventilation using an endotracheal tube. For study purpose, this outcome will be measured at 120 days after randomization. To account for mortality, any death within 120 days post randomization will be counted as "0" mechanical ventilation-free day (worse outcome).

Secondary outcomes

  1. Effects of uc-MSCs on the date of extubation for participants.

    Time frame: From date of randomization until the date of discharge home or date of death from any cause, whichever came first, assessed up to 12 months

    This respiratory outcome will be measured to determine if uc-MSCs have an effect on the date of extubation for participants

  2. Effects of uc-MSCs on the rate of survival without moderate or severe BPD at 36 weeks corrected age.

    Time frame: BPD severity will be assessed for each participant at 36 weeks of corrected age

    This will assess if uc-MSCs impact survival with moderate or severe Bronchopulmonary dysplasia.

  3. Effects of uc-MSCs on the Number of Participants receiving open-label dexamethasone for severe chronic lung disease

    Time frame: From date of randomization until the date of discharge home or date of death from any cause, whichever came first, assessed up to 12 months

    This will help us determine if the intervention reduces use of dexamethasone for the treatment of severe chronic lung disease.

  4. Effects of uc-MSCs on the duration of respiratory support.

    Time frame: From date of randomization until the date of discharge home or date of death from any cause, whichever came first, assessed up to 12 months

    Since ventilation damages underdeveloped lungs, determining the duration of ventilation is important to monitor as an outcome of uc-MSCs.

    We will collect the total number of days on mechanical ventilation, non-invasive ventilation, and oxygen therapy during the NICU hospitalization for each participant

  5. Effects of uc-MSCs on the levels of respiratory support at 36 weeks CA, 40 weeks CA and at hospital discharge.

    Time frame: From date of randomization until the date of discharge home or date of death from any cause, whichever came first, assessed up to 12 months

    The different levels of intensity of ventilation will help us determine if uc-MSCs reduce ventilation days.

    The respiratory support being used are the following (Room air being the least intensive and best for participant health outcome.):

    mechanical ventilation vs. Continuous Positive Airway pressure/High Flow Nasal Canula, vs. Low Flow Nasal Canula vs. room air

  6. Effects of uc-MSCs on the occurrence of pulmonary hypertension related to severe BPD

    Time frame: From date of randomization until the date of discharge home or date of death from any cause, whichever came first, assessed up to 12 months

    This will help us determine the therapeutic properties of UC-MSCs in reducing the occurrence of pulmonary hypertension related to severe BPD.

    Participants will be screened by echocardiography for pulmonary hypertension at 36 weeks of corrected age. Medication for chronic pulmonary hypertension will be collected.

  7. Neurodevelopment and health outcomes at 24 months corrected age (Bayleys)

    Time frame: Assessment will be scheduled at 24 months corrected age.

    The neurodevelopmental assessment will be scheduled at 24 months CA with a window of +/- 6 months (i.e., between 18 to 30 months CA). Participants will be evaluated in the Neonatal Follow-Up clinic for high-risk infants. An assessment of general health and growth will be performed, and the most recent audiology and ophthalmology results will be recorded. The Bayley Scales of Infant Develpment-4th edition will be performed by a qualified professional to further evaluate the neurodevelopment of the participant.

  8. Evaluation of safety of IV administration of UC-MSCs: Dose Limiting toxicity

    Time frame: 24-hours post uc-MSC injection

    Dose-limiting toxicity, defined as one of the following events, occurring within 24-hour post UC-MSC injection:

    • Death
    • Anaphylaxis
    • Increase need for respiratory support define by increase FiO2 >30% from baseline and/or increase in mean airway pressure > 5 cmH2O from baseline
    • Medications to support hemodynamic status (including management of cardiac arrest) including fluid boluses, inotropes, or vasopressors
    • Any SAE not expected in this patient population for which there is no alternative explanation but the IV administration of UC-MSCs and occurring within 1 week after UC-MSCs injection.

  9. Evaluation of safety of IV administration of UC-MSCs: potential adverse event

    Time frame: within 1 week post uc-MSC injection

    Any Serious Adverse Event (SAE) not expected in this patient population for which there is no alternative explanation but the IV administration of UC-MSCs and occurring within 1 week after UC-MSCs injection.

  10. Long-term participant safety follow-up

    Time frame: From enrollment until participant is 10 years of age.

    We plan to have annual parental interviews via telephone until the participant is 10 years old. This is to assess the respiratory status and any new diagnoses of study participants.

  11. Complications of prematurity (assessed at time of hospital discharge)

    Time frame: From date of randomization until the date of discharge home or date of death from any cause, whichever came first, assessed up to 12 months

    The complications of prematurity assessed are the following:

    • BPD.
    • Patent Ductus Arteriosus requiring medical, surgical or device closure.
    • Intra-ventricular hemorrhage ≥ grade 3 according to Papille's classification.
    • Periventricular leukomalacia.
    • Necrotizing enterocolitis ≥ stage 2 according to Bell's classification.
    • Retinopathy of prematurity requiring treatment
    • Late-onset sepsis

Study contacts

Contact information is provided by the study sponsor or research team.

Bernard Thébaud, MD, PhD

CONTACT

[email protected]

613-737-8899 ext. 73905

Chantal Horth

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Ottawa Hospital Research Institute

Other

Collaborators

  • Canadian Institutes of Health Research (CIHR)
  • Stem Cell Network

Registry information

Official study title

Mesenchymal Stromal Cells in Extreme Preterm Infants at Risk of Developing Bronchopulmonary Dysplasia - A Phase 2 Multi-Centre Double Blind Randomized Controlled Trial

Acronym: HULC-2

Important dates

Study start
2025
Primary completion
2028
Study completion
2038
First posted
Jul 10, 2025
Registry last updated
Jul 10, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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