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

NCT03774537 | Unknown Phase 1 DMC

• 1 other identifier: XiaYQ
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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）

Conditions

Bronchopulmonary Dysplasia

Keywords

high risk； Bronchopulmonary Dysplasia；hUC-MSCs

Outcome Measures

Primary Outcomes (1)

• Number of participants with adverse reactions related to infusion after treatment

  To evaluate the safety of hUC-MSCs for BPD.

  24 hours after administration

Secondary Outcomes (6)

• 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

• Changes of high-resolution chest CT in participants

  within 2 years after administration

• Changes of temperature in participants

  3 days after administration

• Changes of blood pressure in participants

  3 days after administration

• Changes of respiratory rate in participants

  3 days after administration

Study Arms (2)

Transplantation of hUC-MSCs

EXPERIMENTAL

Preterm infants at high risk for BPD will receive transplantation of hUC-MSCs.

Drug: Transplantation of hUC-MSCs

No transplantation of hUC-MSCs

OTHER

Preterm infants at high risk for BPD will not receive transplantation of hUC-MSCs

Drug: No transplantation of hUC-MSCs

Interventions

Transplantation of hUC-MSCs DRUG

Preterm infants at high risk for BPD will receive transplantation of hUC-MSCs through intravenous infusion. Dose A - 1 million cells per kg; Dose B - 5 million cells per kg

Also known as: Intravenous infusion of hUC-MSCs

Transplantation of hUC-MSCs

No transplantation of hUC-MSCs DRUG

Preterm infants at high risk for BPD will not receive transplantation of hUC-MSCs

Also known as: No intravenous infusion of hUC-MSCs

No transplantation of hUC-MSCs

Eligibility Criteria

• Age: 4 Days - 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• An infant whose postnatal age is 3 to 14 days, inclusive (for treatment between 5 and 14 days after birth)
• Gestational age is between 23 and 28 weeks (23 weeks ≤ gestational age (GA) \< 28 weeks)
• Birth weight is between 500g and 1000g, inclusive
• 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
• Written consent form signed by a legal representative or a parent.

You may not qualify if:

• 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.
• The participants who have complex congenital heart disease.
• The participants who have severe pulmonary hypertension(cardiac ultrasound confirmed) at the time of assessment.
• 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.
• The participants who have severe chromosome anomalies :Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc).
• The participants who have severe congenital infection(Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc).
• The participants who have severe sepsis or shock.
• The participants who is going to have surgery 72 hours before/after this study drug administration.
• The participants who have surfactant administration within 24 hours before this study drug administration.
• The participants who have severe intracranial hemorrhage ≥ grade 3 or 4.
• The participants who have active pulmonary hemorrhage or active air leak syndrome at the time of assessment.
• The participants who have the history of other clinical studies as a participant.
• The participants who is considered inappropriate by the investigators.

Sponsors & Collaborators

• Children's Hospital of Chongqing Medical University: lead

Study Sites (1)

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, China

RECRUITING

Related Publications (8)

• Chang YS, Ahn SY, Yoo HS, Sung SI, Choi SJ, Oh WI, Park WS. Mesenchymal stem cells for bronchopulmonary dysplasia: phase 1 dose-escalation clinical trial. J Pediatr. 2014 May;164(5):966-972.e6. doi: 10.1016/j.jpeds.2013.12.011. Epub 2014 Feb 6.

  PMID: 24508444 BACKGROUND

• Hayes D Jr, Meadows JT Jr, Murphy BS, Feola DJ, Shook LA, Ballard HO. Pulmonary function outcomes in bronchopulmonary dysplasia through childhood and into adulthood: implications for primary care. Prim Care Respir J. 2011 Jun;20(2):128-33. doi: 10.4104/pcrj.2011.00002.

  PMID: 21336467 BACKGROUND

• Ahn SY, Chang YS, Kim JH, Sung SI, Park WS. Two-Year Follow-Up Outcomes of Premature Infants Enrolled in the Phase I Trial of Mesenchymal Stem Cells Transplantation for Bronchopulmonary Dysplasia. J Pediatr. 2017 Jun;185:49-54.e2. doi: 10.1016/j.jpeds.2017.02.061. Epub 2017 Mar 21.

  PMID: 28341525 BACKGROUND

• Wilson JG, Liu KD, Zhuo H, Caballero L, McMillan M, Fang X, Cosgrove K, Vojnik R, Calfee CS, Lee JW, Rogers AJ, Levitt J, Wiener-Kronish J, Bajwa EK, Leavitt A, McKenna D, Thompson BT, Matthay MA. Mesenchymal stem (stromal) cells for treatment of ARDS: a phase 1 clinical trial. Lancet Respir Med. 2015 Jan;3(1):24-32. doi: 10.1016/S2213-2600(14)70291-7. Epub 2014 Dec 17.

  PMID: 25529339 BACKGROUND

• Laube M, Stolzing A, Thome UH, Fabian C. Therapeutic potential of mesenchymal stem cells for pulmonary complications associated with preterm birth. Int J Biochem Cell Biol. 2016 May;74:18-32. doi: 10.1016/j.biocel.2016.02.023. Epub 2016 Feb 27.

  PMID: 26928452 BACKGROUND

• Pierro M, Ionescu L, Montemurro T, Vadivel A, Weissmann G, Oudit G, Emery D, Bodiga S, Eaton F, Peault B, Mosca F, Lazzari L, Thebaud B. Short-term, long-term and paracrine effect of human umbilical cord-derived stem cells in lung injury prevention and repair in experimental bronchopulmonary dysplasia. Thorax. 2013 May;68(5):475-84. doi: 10.1136/thoraxjnl-2012-202323. Epub 2012 Dec 4.

  PMID: 23212278 BACKGROUND

• Hansmann G, Fernandez-Gonzalez A, Aslam M, Vitali SH, Martin T, Mitsialis SA, Kourembanas S. Mesenchymal stem cell-mediated reversal of bronchopulmonary dysplasia and associated pulmonary hypertension. Pulm Circ. 2012 Apr-Jun;2(2):170-81. doi: 10.4103/2045-8932.97603.

  PMID: 22837858 BACKGROUND

• Yeh TF, Chen CM, Wu SY, Husan Z, Li TC, Hsieh WS, Tsai CH, Lin HC. Intratracheal Administration of Budesonide/Surfactant to Prevent Bronchopulmonary Dysplasia. Am J Respir Crit Care Med. 2016 Jan 1;193(1):86-95. doi: 10.1164/rccm.201505-0861OC.

  PMID: 26351971 BACKGROUND

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

Study Officials

• Zhou Fu

  Children's Hospital of Chongqing Medical University

  STUDY CHAIR

Central Study Contacts

Yunqiu xia

CONTACT

13637719980; sunny_199001@foxmail.com

Lin Zou

CONTACT

18623121280

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director

Study Record Dates

• First Submitted: December 12, 2018
• First Posted: December 13, 2018
• Study Start: March 1, 2019
• Primary Completion: December 1, 2020
• Study Completion: December 31, 2021
• Last Updated: March 6, 2019

Record last verified: 2019-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)