Using stem cells to prevent lung disease in premature infants

The Safety and Feasibility of UMC119-01 Cell Therapy in Infants at High Risk for Bronchopulmonary Dysplasia

This study is testing if giving special stem cells from umbilical cords can help prevent lung disease in premature babies who are at high risk.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 3 Days to 51 Days
• Sex: All
• Sponsor: Meridigen Biotech Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Tainan)
• Trial ID: NCT03631420 on ClinicalTrials.gov

What this trial studies

This clinical study investigates the safety of ex vivo cultured human umbilical cord-derived mesenchymal stem cells in infants at high risk for bronchopulmonary dysplasia (BPD). It is a dose escalation, open-label, single-center study conducted at National Chen-Kung University Hospital. The trial aims to determine the efficacy of this stem cell treatment in preventing BPD in neonatal infants who meet specific eligibility criteria.

Who should consider this trial

Good fit: Ideal candidates for this study are neonatal infants aged 3 to 30 days, born between 23 to 29 weeks of gestation, with a birth weight between 501g to 1249g, and requiring mechanical ventilation.

Not a fit: Patients who are not preterm infants or those who do not require mechanical ventilation may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the incidence of bronchopulmonary dysplasia in premature infants, improving their long-term health outcomes.

How similar studies have performed: While the use of stem cells in treating BPD is a novel approach, similar studies have shown promise in using stem cells for various neonatal conditions.

Eligibility criteria

Inclusion Criteria:

Neonatal infants who fulfil all of the following criteria will be enrolled:

1. Subjects of postnatal age between 3 to 30 days.
2. Are male and female infants born at GA between 23 weeks to 29 weeks. The postmenstrual age of subject received UMC119-01 should be no more than PMA36 weeks.
3. Subjects with birth weight between 501g to 1249 g.
4. Have endotracheal tube in place as part of SoC for preterm infants with BPD at screening and on treatment visit (Day 0), and that they will have not been intubated for the purposes of this study.
5. A subject who is intubated and receiving mechanical ventilation with a fraction of inspired oxygen (FiO2) of 0.25 or greater at Screening.
6. A subject who has had either a deterioration or no change in the setting of mechanical ventilation within the 24 hours before trial enrollment.
7. Written informed consent has been provided by the subject's parents, legal guardians, or a legal representative, who agree to comply with all of the study procedures, including those in the long-term safety surveillance period.

Exclusion Criteria:

Neonatal infants who meet any of the following criteria will be excluded:

1. Have a major congenital abnormality, including neurological (including anencephaly and similar malformations), hepatic, renal, cardiovascular abnormality (except for patent ductus arteriosus, PDA).
2. Have a known genetic syndrome.
3. Have a condition that makes them ineligible for participation in this study, as determined by the investigator.
4. Have C-reactive protein (CRP) \>30 mg/L; or any infections including pneumonia, sepsis, or shock.
5. Have pre-existing severe intraventricular hemorrhage (IVH) (grade ≥3).
6. Have active pulmonary hemorrhage or air leak syndrome.
7. Have abnormal hepatic (AST, ALT \>150 U/L or direct bilirubin \>2 mg/dL or total bilirubin \>15 mg/dL) or renal function (serum creatinine \>1 mg/dL or oliguria).
8. Are known to be infected with HIV or CMV.
9. Are expected to have surgery within 24 hours prior to and/or after UMC119 01 instillation.
10. Are expected to receive any other intratracheal treatments, including surfactant within 72 hours prior to and/or after UMC119 01 instillation.
11. Are currently participating in any other interventional clinical trial.

Where this trial is running

Tainan

• National Chen-Kung University Hospital — Tainan, Taiwan (Recruiting)

Study contacts

• Study coordinator: Claire Liao, MS

Conditions Bronchopulmonary Dysplasia