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

July 11, 2025 updated by: Ottawa Hospital Research Institute

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
9

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Canada
    • Ontario
      • Gloucester, Ontario, Canada, K1T 8L6
        • The Ottawa Hospital - General Campus
      • Toronto, Ontario, Canada, M4N 3M5
        • Sunnybrook Health Sciences Centre

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 week to 3 weeks (Child)

Accepts Healthy Volunteers

No

Description

A participant needs to meet all inclusion criteria between day of life 7-28 to be eligible:

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Mesenchymal Stromal Cell Therapy
Patients 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.
Biological: Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells
Cryopreserved allogeneic umbilical cord tissue-derived mesenchymal stromal cells are thawed and administered intravenously.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Occurrence and rate of dose limiting toxicity
Time Frame: Up to 1 week following uc-MSC injection

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.
Up to 1 week following uc-MSC injection

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Need for Ventilatory Support
Time Frame: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
  • Time to extubation
  • Duration of mechanical ventilation
  • Duration of non-invasive positive pressure respiratory support
  • Duration of supplemental oxygen
From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
Rate of Death
Time Frame: From enrollment until discharge or 40 weeks corrected gestational age (whichever occurs first)
Rate of death until discharge or 40 weeks corrected gestational age, whichever comes first
From enrollment until discharge or 40 weeks corrected gestational age (whichever occurs first)
Occurrence of Other Severe Complications of Prematurity
Time Frame: From enrollment until discharge or 40 weeks corrected gestational age (whichever occurs first)
  • 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
From enrollment until discharge or 40 weeks corrected gestational age (whichever occurs first)
FiO2 and Oxygen Index
Time Frame: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
Measures of gas exchange
From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
Need for Postnatal Steroids
Time Frame: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
This is a yes/no measure
From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
Incidence and Severity of BPD
Time Frame: From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
Measured as mild, moderate, or severe
From enrollment until discharge, 40 weeks corrected gestational age, or death (whichever occurs first)
Rate of Survival Without (moderate or severe) BPD
Time Frame: From enrollment until 36 weeks corrected gestational age
Measured according to the physiological definition of BPD (BPD at 36 weeks corrected age)
From enrollment until 36 weeks corrected gestational age
Changes in Pulmonary Hemodynamics
Time Frame: At enrollment, 48 hours following uc-MSC injection, 28 days of life, and 36 weeks corrected gestational age
Targeted neonatal echocardiography to assess pulmonary hypertension using validated parameters
At enrollment, 48 hours following uc-MSC injection, 28 days of life, and 36 weeks corrected gestational age
Biological Measure of Clinical Improvement
Time Frame: 72-96 hours following uc-MSC injection
Markers of inflammation will be assessed in patient serum samples
72-96 hours following uc-MSC injection
Biological Measure of Lung Improvement
Time Frame: 72-96 hours following uc-MSC injection
Biomarkers of lung improvement will be assessed in patient tracheal aspirate samples
72-96 hours following uc-MSC injection
Long-term Safety Follow-Up
Time Frame: Ten years following follow-up visit
Participant's overall health will be assessed through a questionnaire administered over the phone, once a year for 10 years
Ten years following follow-up visit
Feasibility: Cell Administration
Time Frame: Day of life 7-28
Successful recruitment and administration of cells to nine patients in 18 months
Day of life 7-28
Feasibility: Recruitment Efficiency
Time Frame: Day of life 7-28
  • 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)
Day of life 7-28
Feasibility: Recruitment Timing
Time Frame: Day of life 7-28
  • Median time from screening to enrollment
  • Median time from screening to cell administration
Day of life 7-28
Feasibility: Participant Retainment
Time Frame: From enrollment until follow-up at 18-30 months-of-age
  • Proportion of patients that do not complete cell infusion
  • Proportion of patients enrolled that do not undergo scheduled follow-up
From enrollment until follow-up at 18-30 months-of-age
Bayley Scale of Infant and Toddler Development
Time Frame: 18-30 months-of-age
Assessment of cognitive, language, and motor development
18-30 months-of-age
Animated Information Video
Time Frame: Day of life 7-28
Characterize parental views of an animated MSC information video through brief semi-structured interviews
Day of life 7-28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ottawa Hospital Research Institute

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Canadian Institutes of Health Research (CIHR)
Stem Cell Network
Ontario Institute of Regenerative Medicine (OIRM)

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Bernard Thébaud, MD, PhD, Ottawa Hospital Research Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 17, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 6, 2023

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 6, 2033

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 14, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 3, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 5, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 16, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 11, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • HULC-1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Due to the small sample size, the sharing of individual data has privacy and confidentiality implications.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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