Clinical trial NCT03601416: Human Mesenchymal Stem Cells For Moderate and Severe Bronchopulmonary Dysplasia
Official title: Intravenous Human Umbilical-Cord-Derived Mesenchymal Stem Cells For Moderate and Severe Bronchopulmonary Dysplasias in Children
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Bronchopulmonary Dysplasia
Keywords: human umbilical cord -derived mesenchymal stem cells
moderate and severe bronchopulmonary dysplasia
Lead sponsor: Children's Hospital of Chongqing Medical University
Sponsor class: OTHER
First posted: 2018-07-26
Enrollment: 57
Sex: ALL
Minimum age (years): 28
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 children with moderate and severe bronchopulmonary dysplasia.

Detailed description: Bronchopulmonary dysplasia (BPD) is one of the most common chronic lung diseases with poor prognosis, especially in preterm infants with moderate and severe BPD. However, there is lack of effective therapies for this disease.

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 bronchopulmonary dysplasia.

Eligibility criteria: Inclusion Criteria:

1. Participants with moderate and severe bronchopulmonary dysplasia are not well treated by routine therapy
2. The legal representative or the participant had signed consent.

Exclusion Criteria:

* 1. Severe underlying diseases (e.g. systemic and hematological malignancies, heart failure, liver and kidney failure, immune deficiency, severe infectious diseases, III-IV grade pulmonary hypertension, Lung transplantation, current indications of acute surgery after lung transplantation) 2. Participants whose age is more than 1 year old.

Primary outcomes:
measure: the accumulative duration of oxygen therapy
timeFrame: from the time of diagnosis to the time of stopping oxygen therapy
Secondary outcomes:
measure: Changes of blood pressure in participants
timeFrame: 24 hours after administration
measure: Changes of heart rate in participants
timeFrame: 24 hours after administration
Other outcomes:
measure: Changes of respiratory rate in participants
timeFrame: 24 hours after administration
measure: Changes of high-resolution chest CT in participants
timeFrame: within 2 years after administration
Locations:
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University
state: Chongqing
Study officials:
affiliation: Children's Hospital of Chongqing Medical University
name: Zhou Fu
role: STUDY_CHAIR
References:
citation: 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.
citation: 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.
citation: 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.
citation: Wu X, Xia Y, Zhou O, Song Y, Zhang X, Tian D, Li Q, Shu C, Liu E, Yuan X, He L, Liu C, Li J, Liang X, Yang K, Fu Z, Zou L, Bao L, Dai J. Allogeneic human umbilical cord-derived mesenchymal stem cells for severe bronchopulmonary dysplasia in children: study protocol for a randomized controlled trial (MSC-BPD trial). Trials. 2020 Jan 31;21(1):125. doi: 10.1186/s13063-019-3935-x.