Human Mesenchymal Stem Cells For Infants At High Risk For Bronchopulmonary Dysplasia

Phase 1

• Conditions: Bronchopulmonary Dysplasia
• Interventions: Drug: Transplantation of hUC-MSCs; Drug: No transplantation of hUC-MSCs

• Registration Number: NCT03774537
• Lead Sponsor: Children's Hospital of Chongqing Medical University

Brief Summary

This study is an open-label, single-center, dose escalation study to evaluate of safety and efficacy of human umbilical cord -derived mesenchymal stem cells (hUC-MSCs) in premature infants at high risk for Bronchopulmonary Dysplasia（BPD）

Detailed Description

BPD is a chronic lung disease that occur in premature infants receiving prolonged oxygen pulmonary and ventilator therapy. It remains a main complication of extreme prematurity and currently lacks efficient treatment.The mortality rate of one year after birth is still high and the quality of life is not optimistic.

hUC-MSCs are widely used in clinic due to their low immunogenicity and convenient to get. Many animal study had shown that hUC-MSCs had therapeutic effects on a variety of animal models of lung disease.Furthermore,there are a large number of clinical trials of MSCs applied to various system diseases and the safety was verified.So, the main purpose of this study is to evaluate the safety and efficacy of hUC-MSCs in participants at high risk for BPD

Study Timeline

• Study First Submitted: 2018-12-12
• Study First Submitted QC: 2018-12-12
• Study First Posted: 2018-12-13
• Status Verified: 2019-03
• Study Start: 2019-03-01
• Last Update Submitted: 2019-03-05
• Last Update Posted: 2019-03-06
• Primary Completion: 2020-12-01
• Study Completion: 2021-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. An infant whose postnatal age is 3 to 14 days, inclusive (for treatment between 5 and 14 days after birth)
2. Gestational age is between 23 and 28 weeks (23 weeks ≤ gestational age (GA) < 28 weeks)
3. Birth weight is between 500g and 1000g, inclusive
4. Being intubated and receiving mechanical ventilation within 5-14 days after birth, with a fraction of inspired oxygen (FiO2) of 0.25 or greater at Screening
5. Written consent form signed by a legal representative or a parent.

Exclusion Criteria

1. Although mechanical ventilation or oxygen is required in participants, there are no signs of dyspnea or BPD-related changes in lung imaging, such as central apnea or diaphragm paralysis.
2. The participants who have complex congenital heart disease.
3. The participants who have severe pulmonary hypertension(cardiac ultrasound confirmed) at the time of assessment.
4. The participants who have severe respiratory tract malformation: pierre-robin syndrome, tracheobronchomalacia, vascular ring syndrome, congenital tracheal stenosis, tracheo-esophageal fistula, pulmonary emphysema, pulmonary sequestration, congenital pulmonary dysplasia, congenital pulmonary cyst, congenital spasm, etc.
5. The participants who have severe chromosome anomalies :Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc).
6. The participants who have severe congenital infection(Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc).
7. The participants who have severe sepsis or shock.
8. The participants who is going to have surgery 72 hours before/after this study drug administration.
9. The participants who have surfactant administration within 24 hours before this study drug administration.
10. The participants who have severe intracranial hemorrhage ≥ grade 3 or 4.
11. The participants who have active pulmonary hemorrhage or active air leak syndrome at the time of assessment.
12. The participants who have the history of other clinical studies as a participant.
13. The participants who is considered inappropriate by the investigators.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Transplantation of hUC-MSCs	Transplantation of hUC-MSCs	Preterm infants at high risk for BPD will receive transplantation of hUC-MSCs.
No transplantation of hUC-MSCs	No transplantation of hUC-MSCs	Preterm infants at high risk for BPD will not receive transplantation of hUC-MSCs

Primary Outcome Measures

Name	Time	Method
Number of participants with adverse reactions related to infusion after treatment	24 hours after administration	To evaluate the safety of hUC-MSCs for BPD.

Secondary Outcome Measures

Name	Time	Method
Changes of respiratory rate in participants	3 days after administration	To evaluate the safety of human umbilical cord -derived mesenchymal stem cells for BPD.
Changes of oxygen saturation in participants	3 days after administration	To evaluate the safety of human umbilical cord -derived mesenchymal stem cells for BPD.
Changes of high-resolution chest CT in participants	within 2 years after administration	To evaluate the safety and efficacy of human umbilical cord -derived mesenchymal stem cells for BPDmesenchymal stem cells for BPD.
The incidence and severity of BPD defined by the National Institutes of Child Health and Human Development (NICHD) workshop.	at the corrected gestational age of 36 weeks	To evaluate the efficacy of hUC-MSCs to prevent preterm infants at high risk of BPD from developing BPD
Changes of temperature in participants	3 days after administration	To evaluate the safety of human umbilical cord -derived mesenchymal stem cells for BPD.mesenchymal stem cells for BPD.
Changes of blood pressure in participants	3 days after administration	To evaluate the safety of human umbilical cord -derived mesenchymal stem cells for BPD. Blood pressure is measured by electronic sphygmomanometer.

Trial Locations

Locations (1)

Children's Hospital of Chongqing Medical University; 🇨🇳 Chongqing, Chongqing, China