Human Mesenchymal Stem Cells For Bronchopulmonary Dysplasia

NCT ID: NCT03558334

Last Updated: 2019-05-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-28

Study Completion Date

2022-06-30

Brief Summary

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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).

Detailed Description

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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 with moderate and severe BPD.

Conditions

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Bronchopulmonary Dysplasia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Transplantation of Mesenchymal Stem Cell

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

Group Type EXPERIMENTAL

Transplantation of mesenchymal stem cell

Intervention Type 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

No Transplantation of Mesenchymal Stem Cell

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

Group Type ACTIVE_COMPARATOR

No transplantation of mesenchymal stem cell

Intervention Type DRUG

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

Interventions

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Transplantation of mesenchymal stem cell

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

Intervention Type DRUG

No transplantation of mesenchymal stem cell

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

Intervention Type DRUG

Other Intervention Names

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Intravenous infusion of mesenchymal stem cell No intravenous infusion of mesenchymal stem cell

Eligibility Criteria

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Inclusion Criteria

1. The participants meet the diagnostic criteria for moderate and severe BPD established by the National Institutes of Child Health and Human Development (NICHD) workshop.
2. The participants have abnormal respiratory manifestations.
3. 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.
Minimum Eligible Age

28 Days

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Children's Hospital of Chongqing Medical University

OTHER

Sponsor Role lead

Responsible Party

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Xia Yunqiu

doctor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Zhou Fu

Role: STUDY_CHAIR

Children's Hospital of Chongqing Medical University

Locations

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Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Yunqiu Xia

Role: CONTACT

13637719980

Lin Zou

Role: CONTACT

18623121280

Facility Contacts

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Yunqiu Xia

Role: primary

13637719980

References

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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.

Reference Type BACKGROUND
PMID: 24508444 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21336467 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 28341525 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 25529339 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 26928452 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 23212278 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 22837858 (View on PubMed)

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.

Reference Type DERIVED
PMID: 37223507 (View on PubMed)

Other Identifiers

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XYunqiu

Identifier Type: -

Identifier Source: org_study_id

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