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Bronchopulmonary DysplasiaPhase 1NCT04255147

Cellular Therapy for Extreme Preterm Infants at Risk of Developing Bronchopulmonary Dysplasia

A Phase 1 study testing Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells in people with Bronchopulmonary Dysplasia, run by Ottawa Hospital Research Institute at 2 sites in Canada.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Bronchopulmonary dysplasia (BPD) is a common and chronic lung disease that occurs in preterm infants following ventilator and oxygen therapy and is associated with long-term health consequences. Read more about this study

Who is it for?
Ages 7 to 28 days
Conditions the study is for
Bronchopulmonary Dysplasia
What is being tested?
Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
2 sitesCanada
Study dates
17 Oct 2022to 6 Nov 2033
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 7 to 28 days
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Admission to The Ottawa Hospital (TOH) NICU - General Campus or Sunnybrook Health Sciences Centre NICU
  • Gestational age at birth < 28 weeks
  • Intubated on mechanical ventilation
  • Fraction of inspired oxygen ≥ 30%
  • Parents or substitute decision make must provide written informed consent

Who cannot join

  • Severe congenital anomaly by antenatal ultrasound and physical examination
  • Ongoing shock and severe sepsis (confirmed by positive blood or cerebrospinal fluid culture) as per attending physician
  • Severe pulmonary hemorrhage
  • Active pneumothorax (with chest tube in-situ)
  • Hemodynamically significant PDA
  • Participants with caregiver unable to speak English or French
  • Patient i moribund, not expected to survive
  • Planned to be extubated in the 24 hours after uc-MSC administration

Trial locations and contacts

2 sites in 1 country.

Canada2 sites

  • The Ottawa Hospital - General Campus

    Gloucester, Ontario, K1T 8L6

  • Sunnybrook Health Sciences Centre

    Toronto, Ontario, M4N 3M5

Other trials in Canada

About this study

Official title: Helping Underdeveloped Lungs With Cells (HULC): Mesenchymal Stromal Cells in Extreme Preterm Infants at Risk of Developing Bronchopulmonary Dysplasia - Phase 1 Study

Bronchopulmonary dysplasia (BPD) is a common and chronic lung disease that occurs in preterm infants following ventilator and oxygen therapy and is associated with long-term health consequences. Preclinical research shows that mesenchymal stromal cells (MSCs) can modify a number of pathophysiological processes that are central to the progression of BPD and thus present as a promising new treatment option. The main purpose of this Phase I study is to evaluate the safety of human umbilical cord tissue-derived MSCs in extremely preterm infants at risk of developing BPD.

Read the detailed description

Complications of extreme preterm birth are the primary cause of mortality in children under the age of five. Bronchopulmonary dysplasia (BPD), the chronic lung disease that follows ventilator and oxygen therapy for acute respiratory failure, is the most common complication of extreme prematurity and contributes to life-long respiratory and neurological impairment. Currently, there is no effective treatment for BPD. The multi-factorial nature of BPD makes it challenging for traditional pharmacological therapies targeting a single pathway to have a major impact on outcome. Mesenchymal stromal cells (MSCs) may provide a promising new treatment avenue due to their pleiotropic effects that may prevent neonatal lung injury while promoting lung (and other organ) growth. A systematic review and meta-analysis of all preclinical studies testing MSCs in neonatal lung injury models provides strong evidence for the lung protective effect of MSCs. Additionally, studies in a large preclinical model of extreme prematurity and chronic lung injury suggest feasibility, safety and short-term hemodynamic benefit of intravenously delivered human umbilical cord tissue-derived MSCs (uc-MSC).

The aim of this study is to establish the safety, maximum feasible dose and feasibility of intravenously delivered allogeneic uc-MSCs in preterm infants at risk of developing BPD. This will be a Phase 1, open-label, single center, dose-escalating trial using a 3+3+3 design.

Being tested
Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells
Keywords
Phase I clinical trial, Stem cells, Mesenchymal stromal cell, Bronchopulmonary Dysplasia, Preterm birth, Cell therapy
Medical subject headings
Bronchopulmonary Dysplasia, Premature Birth

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal CellsBiological

    Cryopreserved allogeneic umbilical cord tissue-derived mesenchymal stromal cells are thawed and administered intravenously.

Study groups in NCT04255147
Study groupRoleWhat they receive
Mesenchymal Stromal Cell TherapyExperimentalPatients are enrolled into one of three escalating dose panels based on the time of enrolment. The first three patients will receive 1 million cells/kg of body weight, the next three patients will receive 3 million cells/kg of body weight, and the final three patients will receive 10 million cells/kg of body weight. Progression through the escalating dose panels is subject to review by an independent Data Safety Monitoring Committee.
Receives: Biological: Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Occurrence and rate of dose limiting toxicity

    Dose limiting toxicity consists of the following events:

    • Death occurring within 24 hours of injection;
    • Pulmonary embolism defined as acute increase in right ventricular afterload (identified by serial targeted neonatal echocardiography) and signs of acute increased dead space ventilation (respiratory distress, increased PaCO2, increased minute ventilation) occurring within 24 hours of injection;
    • Hypersensitivity / anaphylactic to uc-MSCs defined as any severe systemic inflammatory response syndrome with negative blood culture not consistent with the overall clinical course of the infant occurring within 72 hours of injection;
    • Any other serious adverse event not expected in this patient population for which there is no alternative explanation but the administration of uc-MSCs, occurring within 1 week of injection.

    Measured: Up to 1 week following uc-MSC injection

Secondary outcomes (17)

  1. Rate of Death

    Rate of death until discharge or 40 weeks corrected gestational age, whichever comes first

    Measured: From enrollment until discharge or 40 weeks corrected gestational age (whichever occurs first)

  2. Occurrence of Other Severe Complications of Prematurity

    • Blood culture-proven sepsis
    • Patent ductus arteriosus (treated medically or surgically)
    • Necrotizing enterocolitis
    • Isolated intestinal perforation
    • Retinopathy of prematurity requiring treatment
    • Severe intraventricular hemorrhage (≥ grade 3)
    • Cystic periventricular leukomalacia

    Measured: From enrollment until discharge or 40 weeks corrected gestational age (whichever occurs first)

  3. FiO2 and Oxygen Index

    Measures of gas exchange

    Measured: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)

  4. Need for Ventilatory Support

    • Time to extubation
    • Duration of mechanical ventilation
    • Duration of non-invasive positive pressure respiratory support
    • Duration of supplemental oxygen

    Measured: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)

  5. Need for Postnatal Steroids

    This is a yes/no measure

    Measured: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)

  6. Incidence and Severity of BPD

    Measured as mild, moderate, or severe

    Measured: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)

  7. Rate of Survival Without (moderate or severe) BPD

    Measured according to the physiological definition of BPD (BPD at 36 weeks corrected age)

    Measured: From enrollment until 36 weeks corrected gestational age

  8. Changes in Pulmonary Hemodynamics

    Targeted neonatal echocardiography to assess pulmonary hypertension using validated parameters

    Measured: At enrollment, 48 hours following uc-MSC injection, 28 days of life, and 36 weeks corrected gestational age

  9. Biological Measure of Clinical Improvement

    Markers of inflammation will be assessed in patient serum samples

    Measured: 72-96 hours following uc-MSC injection

  10. Biological Measure of Lung Improvement

    Biomarkers of lung improvement will be assessed in patient tracheal aspirate samples

    Measured: 72-96 hours following uc-MSC injection

  11. Feasibility: Cell Administration

    Successful recruitment and administration of cells to nine patients in 18 months

    Measured: Day of life 7-28

  12. Feasibility: Recruitment Efficiency

    • Proportion of potentially eligible patients that are successfully screened
    • Proportion of participants successfully screened who do not enroll (reason for failure to enroll will be recorded)

    Measured: Day of life 7-28

  13. Feasibility: Recruitment Timing

    • Median time from screening to enrollment
    • Median time from screening to cell administration

    Measured: Day of life 7-28

  14. Feasibility: Participant Retainment

    • Proportion of patients that do not complete cell infusion
    • Proportion of patients enrolled that do not undergo scheduled follow-up

    Measured: From enrollment until follow-up at 18-30 months-of-age

  15. Bayley Scale of Infant and Toddler Development

    Assessment of cognitive, language, and motor development

    Measured: 18-30 months-of-age

  16. Long-term Safety Follow-Up

    Participant's overall health will be assessed through a questionnaire administered over the phone, once a year for 10 years

    Measured: Ten years following follow-up visit

  17. Animated Information Video

    Characterize parental views of an animated MSC information video through brief semi-structured interviews

    Measured: Day of life 7-28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Feb 2020
  2. Study start17 Oct 2022
  3. Primary completion6 Nov 2023
  4. Study completion6 Nov 2033 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04255147
Sponsor's protocol ID
HULC-1
Status last verified
Jul 2025
First submitted
14 Jan 2020
Last updated
16 Jul 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: Due to the small sample size, the sharing of individual data has privacy and confidentiality implications.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells in people with Bronchopulmonary Dysplasia, run by Ottawa Hospital Research Institute at 2 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Occurrence and rate of dose limiting toxicity (time frame: Up to 1 week following uc-MSC injection). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04255147.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.

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