Mesenchymal Stem Cells for The Treatment of Bronchopulmonary Dysplasia in Infants

• ClinicalTrials.gov Identifier: NCT03631420
• Recruitment Status: Recruiting
• First Posted: August 15, 2018
• Last Update Posted: December 19, 2019
• Sponsor: Meridigen Biotech Co., Ltd.
• Information provided by (Responsible Party): Meridigen Biotech Co., Ltd.

Study Description

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 with servere BPD.

Condition or disease	Intervention/treatment	Phase
Bronchopulmonary Dysplasia	Biological: Human Umbilical Cord Derived-Mesenchymal Stem Cells	Phase 1

Detailed Description:

UMC119-01 is ex vivo cultured human umbilical cord tissue-derived mensenchymal stem cells product which is intended for treatment of Bronchopulmonary dysplasia.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 9 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: The Safety and Feasibility of UMC119-01 Cell Therapy in Infants With Bronchopulmonary Dysplasia
• Actual Study Start Date: October 26, 2018
• Estimated Primary Completion Date: October 31, 2020
• Estimated Study Completion Date: October 31, 2022

Arms and Interventions

Arm	Intervention/treatment
Experimental: UMC119-01; UMC119-01 is ex vivo cultured human umbilical cord tissue-derived mensenchymal stem cells product	Biological: Human Umbilical Cord Derived-Mesenchymal Stem Cells; Cohort 1 : 3 million cells/kg ; Cohort 2 : 10 million cells/kg ; Cohort 3 : 30 million cells/kg

Outcome Measures

Primary Outcome Measures :

1. The incidence and frequency of adverse events related to administration of UMC119-01. [ Time Frame: 3 months from the day of administration ]
   • The incidence and frequency of adverse events (AEs) and serious adverse events (SAEs), including their relationship to the administration of UMC119 01.

Secondary Outcome Measures :

1. The percentage of subjects who died from any cause during the study. [ Time Frame: 3 months from the day of administration ]
   The percentage of subjects who died from any cause during the study.
2. Changes of the oxygen saturation. [ Time Frame: 3 months from the day of administration ]
   Changes from baseline of the oxygen saturation.
3. Changes of chest x-ray findings in participants before and after administration. [ Time Frame: 3 months from the day of administration ]
   Changes from baseline to 3 months of chest x-ray findings in participants before and after administration.
4. Changes of inflammatory markers (pg/ml) before and after administration. [ Time Frame: 3 days and 7 days after administration ]
   Changes from baseline of inflammatory markers (pg/ml) .
5. Comparison of the incidence and severity of preterm children's complications in participants before and after administration. [ Time Frame: 3 months from the day of administration ]
   Comparison of the incidence and severity of preterm children's complications in participants before and after administration.
6. The number of days of intubation, or where ventilator or oxygen therapy. [ Time Frame: 3 months from the day of administration ]
   The number of days of intubation, or where ventilator or oxygen therapy.

Eligibility Criteria

• Ages Eligible for Study: 36 Weeks to 48 Weeks (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

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

1. Are male and female infants born at a GA <29 weeks, with a birth weight ≤1250 g.
2. Have been diagnosed with severe BPD in accordance with National Institute of Child Health and Human Development (NICHD) criteria which require that at 36 weeks PMA there is need for 30% oxygen and/or positive pressure (positive-pressure ventilation or nasal continuous positive airway pressure).

3. On Day 1 (which will occur at, but not more than 12 weeks following, 36 weeks PMA), required oxygen for at least 28 days with no sign of weaning from the following respiratory support:

   FiO2 ≥30% and/or positive pressure (positive-pressure ventilation or nasal continuous positive airway pressure).

4. Have endotracheal tube in place as part of SoC for preterm infants with BPD at screening and on Day 1 and that they will have not been intubated for the purposes of this study.
5. 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, or cardiovascular abnormality (except for 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, within 1 week prior to enrolment of this study.
5. Have pre-existing severe IVH (grade ≥3).
6. Have active pulmonary hemorrhage or air leak syndrome within 1 week prior to enrolment into the study.
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 72 hours prior to and/or after UMC119 01 instillation.
10. Are expected to receive any other intratracheal treatments, including surfactant or nitric oxide within 72 hours prior to and/or after UMC119 01 instillation.
11. Are currently participating in any other interventional clinical trial.

Contacts and Locations

Contacts

Contact: Katherine Huang, MS; +886-2-26275175 ext 19926; Katherine.Huang@meridigen.com

Locations

Taiwan

National Chen-Kung University Hospital; Recruiting

Tainan City, Taiwan

Contact: Katherine Huang, MS +886-2-2627-5175 ext 19926 Katherine.Huang@meridigen.com

Principal Investigator: Yuh-Jyh Lin, MD

Sub-Investigator: Yung-Chieh Lin, MD

Sub-Investigator: Yu-Shan Chang, MD

Sub-Investigator: Yen-Ju Chen, MD

Sponsors and Collaborators

Meridigen Biotech Co., Ltd.

More Information

• Responsible Party: Meridigen Biotech Co., Ltd.
• ClinicalTrials.gov Identifier: NCT03631420
• Other Study ID Numbers: UMC119-01-001
• First Posted: August 15, 2018
• Last Update Posted: December 19, 2019
• Last Verified: December 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Bronchopulmonary Dysplasia; Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases