Cellular Therapy for Bronchopulmonary Dysplasia

Recruiting at 2 trial locations

Overseen By Study Coordinator

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: Ottawa Hospital Research Institute

Disqualifiers: Congenital anomaly, Severe sepsis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 1 week following uc-MSC injection

9 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications.

What data supports the effectiveness of the treatment Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells for Bronchopulmonary Dysplasia?

Research suggests that mesenchymal stem cells (MSCs) from umbilical cords have shown promise in improving lung injuries in conditions similar to bronchopulmonary dysplasia (BPD), such as in animal studies where they helped reduce lung damage. Additionally, preliminary outcomes in infants with BPD indicate that this treatment is safe and may support lung development. ^{1 2 3 4 5}

Is cellular therapy using umbilical cord-derived mesenchymal stem cells safe for humans?

Studies have shown that using umbilical cord-derived mesenchymal stem cells in humans, particularly for bronchopulmonary dysplasia, is generally safe. In clinical trials, no serious side effects related to the treatment were observed, and it was well-tolerated by patients. ^{2 3 4 6 7}

How is the treatment with umbilical cord-derived mesenchymal stromal cells different from other treatments for bronchopulmonary dysplasia?

This treatment uses stem cells from umbilical cords, which are special cells that can help repair and improve lung function in premature infants with bronchopulmonary dysplasia. Unlike other treatments, it involves administering these cells intravenously (through a vein) to potentially heal lung damage and improve breathing. ^{1 2 3 5 7}

What is the purpose of this trial?

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.

Research Team

Bernard Thébaud, MD, PhD

Principal Investigator

Ottawa Hospital Research Institute

Eligibility Criteria

This trial is for extremely preterm infants born before 28 weeks who are on mechanical ventilation with a high need for oxygen. They must be admitted to specific NICUs and have parents or guardians able to consent. Infants with severe birth defects, active lung complications, heart issues, ongoing shock or sepsis, language barriers in caregivers, expected extubation within 24 hours after treatment, or those not likely to survive are excluded.

Inclusion Criteria

Admission to The Ottawa Hospital (TOH) NICU - General Campus or Sunnybrook Health Sciences Centre NICU

You were born before 28 weeks of pregnancy.

I am currently on a breathing machine.

Exclusion Criteria

I have a significant heart defect known as PDA.

I am currently experiencing severe shock and infection, confirmed by tests.

I have had severe bleeding in my lungs.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Day of life 7-28

Treatment

Participants receive intravenously delivered allogeneic uc-MSCs in a dose-escalating manner

Up to 1 week following uc-MSC injection

Multiple visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

From enrollment until discharge, 40 weeks corrected gestational age, or death

Long-term Safety Follow-Up

Participant's overall health will be assessed through a questionnaire administered over the phone

Ten years following follow-up visit

Treatment Details

Interventions

• Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells

Trial Overview The study tests the safety of using cells from umbilical cord tissue (called mesenchymal stromal cells) in very early-born babies at risk of developing a chronic lung disease known as Bronchopulmonary Dysplasia. It's an initial phase trial aimed at seeing if this new therapy is safe for these vulnerable infants.

Participant Groups

Group I: Mesenchymal Stromal Cell Therapy Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Ottawa Hospital Research Institute

Lead Sponsor

Trials

585

Recruited

3,283,000+

Canadian Institutes of Health Research (CIHR)

Collaborator

Trials

1,417

Recruited

26,550,000+

Ontario Institute of Regenerative Medicine (OIRM)

Collaborator

Trials

1

Recruited

9+

Stem Cell Network

Collaborator

Trials

9

Recruited

600+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Dose-dependent effects of human umbilical cord-derived mesenchymal stem cell treatment in hyperoxia-induced lung injury of neonatal rats. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Allogeneic administration of human umbilical cord-derived mesenchymal stem/stromal cells for bronchopulmonary dysplasia: preliminary outcomes in four Vietnamese infants. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Allogeneic human umbilical cord-derived mesenchymal stem cells for severe bronchopulmonary dysplasia in children: study protocol for a randomized controlled trial (MSC-BPD trial). [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Intratracheal transplantation of mesenchymal stem cells simultaneously attenuates both lung and brain injuries in hyperoxic newborn rats. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cells protect against neonatal rat hyperoxic lung injury. [2018]

6. Netherlands pubmed.ncbi.nlm.nih.gov

Phase I trial of human umbilical cord-derived mesenchymal stem cells for treatment of severe bronchopulmonary dysplasia. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Mesenchymal Stromal Cell Therapy for Respiratory Complications of Extreme Prematurity. [2019]