Safety and Efficacy of Umbilical Cord-derived Mesenchymal Stem Cell Transplantation in the Treatment of Bronchopulmonary Dysplasia in Premature Infants
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Extubating time after MSC transplantation
Study Overview
Brief Summary
Bronchopulmonary dysplasia (BPD) is a chronic lung disease, which is a major complication of very low and ultra-low preterm infants. Moderate and severe BPD survivors are prone to adverse outcomes such as impaired lung function, childhood exercise intolerance, and neurodevelopmental retardation in the long term, which seriously affects their quality of life and brings a heavy burden to society and families. However, the pathogenesis of BPD is complex, including pulmonary vascular dysplasia, lung inflammation, and impaired alveolar development. There is currently no specific clinical drug to cure BPD. Mesenchymal stem cells (MSCs) are a kind of multipotent stem cells that exist in almost all organs and tissues of individuals. MSCs have the properties including self-renewal, multi-directional differentiation, and immunosuppressive and anti-inflammatory abilities. Preclinical studies have shown that MSCs can alleviate BPD by improving alveolar and pulmonary vascular development, and reducing pulmonary fibrosis. Several phase I clinical studies have demonstrated that intratracheal transplantation of human umbilical cord blood-derived mesenchymal stem cells for children with BPD is safe and feasible.
This study aims to further evaluate the safety and efficacy of umbilical cord-derived mesenchymal stem cell transplantation in the treatment of severe BPD in premature infants, in the hope of increasing the survival rate and improving the prognosis of severe BPD.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 36 Weeks to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •23-29 weeks of gestation, birth weight 500-1500g;
- •For patients with no improvement or aggravation of lung condition after DART hormone therapy, and positive pressure ventilation by tracheal intubation is still required at a correct gestational age of 36 weeks.
- •Children with severe BPD after early use of PS
- •Parents agree to participate in clinical trials.
Exclusion Criteria
- •Premature infants not suitable for the given gestational age;
- •Other congenital structural malformations of trachea, bronchus and lungs;
- •Complicated with severe congenital heart disease;
- •Complicated with Periventricular Leukomalacia (PVL);
- •Complicated with intraventricular hemorrhage (IVH) above level 3;
- •Septic shock or positive blood culture;
- •Acute pulmonary hemorrhage;
- •Intracranial and extracranial diseases affecting respiratory rate and rhythm.
Arms & Interventions
MSC transplantation
MSCs (1×10^7/kg) are administered intratracheally to participators.
Intervention: MSC (Drug)
Outcomes
Primary Outcomes
Extubating time after MSC transplantation
Time Frame: until 24 months of corrected age
Record how long it takes for the participants' tracheal tubes to be removed after MSC transplantation.
The number of times and total duration of re-intubation after extubating
Time Frame: until 24 months of corrected age
If the participants' tracheal tubes are removed after MSC transplantation, record the number of times and total duration of tracheal re-intubation until discharge and 24 months of corrected age.
Mortality of BPD
Time Frame: until 24 months of corrected age
Record the number of participants who died from BPD.
Secondary Outcomes
- Further assess the severity of BPD by detecting the levels of pro-inflammatory cytokine in alveolar lavage fluid, pulmonary function index and chest radiography.(until 24 months of corrected age)
- Short-term safety assessment of MSC transplantation by whether anaphylaxis and severe infection are observed within 24 hours after MSC transplantation.(within 24 hours after MSC transplantation)
- long-term safety assessment of MSC transplantation by whether intraventricular hemorrhage, periventricular leukomalacia, neurodevelopmental problems and tumor formation are observed after MSC transplantation until 24 months of corrected age.(until 24 months of corrected age)
Investigators
Shu Qiang
Professor of Pediatrics, Chief surgeon, Principal Investigator, Hospital secretary of Party Committee, Children's Hospital, Zhejiang University School of Medicine
The Children's Hospital of Zhejiang University School of Medicine