Human Mesenchymal Stem Cells For Bronchopulmonary Dysplasia

NCT03558334 | Unknown Phase 1 DMC

• 1 other identifier: XYunqiu
• Study Type: interventional
• Target: 12
• 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 for moderate and severe Bronchopulmonary Dysplasia（BPD）.

Conditions

Bronchopulmonary Dysplasia

Keywords

Bronchopulmonary Dysplasia; human umbilical cord -derived mesenchymal stem cells

Outcome Measures

Primary Outcomes (1)

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

  To evaluate the safety of human umbilical cord -derived mesenchymal stem cells for BPD.

  24 hours after administration

Secondary Outcomes (7)

• 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 heart rate in participants

  3 days after administration

• Changes of respiratory rate in participants

  3 days after administration

Study Arms (2)

Transplantation of Mesenchymal Stem Cell

EXPERIMENTAL

Mesenchymal stem cell will be given to preterm infants with BPD.

Drug: Transplantation of mesenchymal stem cell

No Transplantation of Mesenchymal Stem Cell

ACTIVE COMPARATOR

Mesenchymal stem cell will be not given to preterm infants with BPD.

Drug: No transplantation of mesenchymal stem cell

Interventions

Transplantation of mesenchymal stem cell DRUG

Human umbilical cord-derived mesenchymal stem cell will be given to preterm infants through intravenous infusion. Dose A - 1 million cells per kg Dose B - 5 million cells per kg

Also known as: Intravenous infusion of mesenchymal stem cell

Transplantation of Mesenchymal Stem Cell

No transplantation of mesenchymal stem cell DRUG

Human umbilical cord-derived mesenchymal stem cell will be not given to preterm infants through intravenous infusion.

Also known as: No intravenous infusion of mesenchymal stem cell

No Transplantation of Mesenchymal Stem Cell

Eligibility Criteria

• Age: 28 Days+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• The participants meet the diagnostic criteria for moderate and severe BPD established by the National Institutes of Child Health and Human Development (NICHD) workshop.
• The participants have abnormal respiratory manifestations.
• 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, 400014, 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

• Xia Y, Lang T, Niu Y, Wu X, Zhou O, Dai J, Bao L, Yang K, Zou L, Fu Z, Geng G. Phase I trial of human umbilical cord-derived mesenchymal stem cells for treatment of severe bronchopulmonary dysplasia. Genes Dis. 2022 Feb 22;10(2):521-530. doi: 10.1016/j.gendis.2022.02.001. eCollection 2023 Mar.

  PMID: 37223507 DERIVED

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: doctor

Study Record Dates

• First Submitted: June 4, 2018
• First Posted: June 15, 2018
• Study Start: June 28, 2018
• Primary Completion: December 31, 2021
• Study Completion: June 30, 2022
• Last Updated: May 21, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)