Mesenchymal Stem Cells for Prevention of Bronchopulmonary Dysplasia in Infants

NCT03631420 | Recruiting Phase 1 FDA Drug

• 1 other identifier: UMC119-01-001
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

The clinical study with UMC119-01 is designed to investigate the safety in patients with bronchopulmonary dysplasia ("BPD"). This will be a dose escalation, open-label, single-center study in infants at high risk for BPD.

Conditions

Bronchopulmonary Dysplasia

Outcome Measures

Primary Outcomes (1)

• The incidence and frequency of adverse events related to administration of UMC119-01.

  • The incidence and frequency of adverse events (AEs) and serious adverse events (SAEs), including their relationship to the administration of UMC119 01.

  3 months from the day of administration

Secondary Outcomes (7)

• The percentage of subjects who died from any cause during the study.

  3 months from the day of administration

• Changes of the oxygen saturation.

  3 months from the day of administration

• Changes of chest x-ray findings in participants before and after administration.

  3 months from the day of administration

• Changes of inflammatory markers (pg/ml) before and after administration.

  3 days and 7 days after administration

• Comparison of the incidence and severity of preterm children's complications in participants before and after administration.

  3 months from the day of administration

Study Arms (1)

UMC119-01

EXPERIMENTAL

UMC119-01 is ex vivo cultured human umbilical cord tissue-derived mensenchymal stem cells product

Biological: Human Umbilical Cord Derived-Mesenchymal Stem Cells

Interventions

Human Umbilical Cord Derived-Mesenchymal Stem Cells BIOLOGICAL

Cohort 1 : 3 million cells/kg ; Cohort 2 : 10 million cells/kg ; Cohort 3 : 30 million cells/kg

UMC119-01

Eligibility Criteria

• Age: 3 Days - 51 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Neonatal infants who fulfil all of the following criteria will be enrolled:
• Subjects of postnatal age between 3 to 30 days.
• 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.
• Subjects with birth weight between 501g to 1249 g.
• 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.
• A subject who is intubated and receiving mechanical ventilation with a fraction of inspired oxygen (FiO2) of 0.25 or greater at Screening.
• A subject who has had either a deterioration or no change in the setting of mechanical ventilation within the 24 hours before trial enrollment.
• 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.

You may not qualify if:

• Neonatal infants who meet any of the following criteria will be excluded:
• Have a major congenital abnormality, including neurological (including anencephaly and similar malformations), hepatic, renal, cardiovascular abnormality (except for patent ductus arteriosus, PDA).
• Have a known genetic syndrome.
• Have a condition that makes them ineligible for participation in this study, as determined by the investigator.
• Have C-reactive protein (CRP) \>30 mg/L; or any infections including pneumonia, sepsis, or shock.
• Have pre-existing severe intraventricular hemorrhage (IVH) (grade ≥3).
• Have active pulmonary hemorrhage or air leak syndrome.
• 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).
• Are known to be infected with HIV or CMV.
• Are expected to have surgery within 24 hours prior to and/or after UMC119 01 instillation.
• Are expected to receive any other intratracheal treatments, including surfactant within 72 hours prior to and/or after UMC119 01 instillation.
• Are currently participating in any other interventional clinical trial.

Sponsors & Collaborators

• Meridigen Biotech Co., Ltd.: lead

Study Sites (1)

National Chen-Kung University Hospital

Tainan, Taiwan

RECRUITING

MeSH Terms

Conditions

Bronchopulmonary Dysplasia

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Claire Liao, MS

CONTACT

+886-2-8978-7777; claire.liao@meridigen.com

Joseph Chen

CONTACT

+886-2-8978-7777; joseph.chen@meridigen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2018
• First Posted: August 15, 2018
• Study Start: October 26, 2018
• Primary Completion: December 31, 2025
• Study Completion (Estimated): July 31, 2027
• Last Updated: December 4, 2023

Record last verified: 2022-12

Locations

TW (1)