Bronchopulmonary Dysplasia
Conditions
Keywords
Bronchopulmonary dysplasia, Preterm newbnorn, Mesenchymal stem cells
Brief summary
Bronchopulmonary dysplasia (BPD) is a disease that affects preterm newborn patients, preventing their lungs from developing properly. Allogeneic fetal stem mesenchymal cells from umbilical cord could reduce the prevalence of BPD in this patients.
Detailed description
Bronchopulmonary dysplasia (BPD) is a disease that affects preterm newborn patients, preventing their lungs from developing properly, and it is a disease that is nowadays increasing due to the improvement in the survival of this patients (affecting 15-50% of them). In the Fase I Clinical Trial, the use of allogeneic fetal stem mesenchymal cells from umbilical cord proved to be safe, with no mortality or Adverse Events reported. The Fase II Clinical Trial is based in the hypothesis that the administation of mesenchymal stem cells is not only safe but feasible and can help reducing the chance of a preterm newborn patient developing BPD.
Interventions
BIOLOGICALControl
Standard treatment
BIOLOGICALAllogenic fetal mesenchymal stem cells from umbilical cord - three infusions
3 doses of 5 million MSC will be administered
BIOLOGICALAllogenic fetal mesenchymal stem cells from umbilical cord - six infusions
6 doses of 5 million MSC will be administered
Sponsors
Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Parallel Group Assignment
Eligibility
Sex/Gender
ALL
Age
1 Months to 28 Weeks
Healthy volunteers
No
Inclusion criteria
* Alive newborns weighing ≤ 1250 grams and GA ≤ 28 weeks, who are on mechanical ventilation with a FiO2 ≥0.3 between days 5 and 14 of life, with no immediate extubation foreseeable.
Exclusion criteria
* Presence of another concomitant congenital pathology at the time of inclusion: pulmonary malformations with compromised pulmonary function, active pulmonary haemorrhage, severe pulmonary hypoplasia, renal malformations with systemic compromise, congenital heart disease, polymalformative syndromes, chromosomopathies. * Presence of refractory haemodynamic instability of any cause at the time of inclusion. * Presence of severe neurological damage at the time of inclusion (HIV grade III or higher). * Patients who have required major surgery in the 72 hours prior to inclusion. * Patients who have necrotising enterocolitis (NEC) grades ≥II at the time of inclusion, according to the Bell classification. * Patients who are children of a mother with HIV
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Security of MSC therapy in very low birth weight preterm babies at risk of developing bronchopulmonary dysplasia | 24 months | Number of patients with adverse events during the infusion time and during all study; and comorbilities due to preterm birth. |
| feasibility variable | 24 months | Number of days of life from birth to administration of the first dose and number of days of life in successive doses. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Exitus on week 36 and 40 of post-menstrual age or at hospital discharge | 24 months | (Yes/No) |
| Incidence of comorbidities resulting from prematurity from the time of screening to 40 weeks' EPM, hospital discharge or death. | 24 months | (sepsis confirmed by blood culture, treated patent ductus arteriosus, non-pharmacological ductal closure, necrotising enterocolitis, isolated bowel perforation, intraventricular haemorrhage ≥ 2, retinopathy ≥ grade 2) |
| Biomarker analysis (IL-1beta, IL-6, IL8, TGF beta, TNF alfa, GM-CSF, NLRP3, RAGE, HMGB1, VEGF, HGF, GREMLIN1, sVEGFR1, SP-D, SMPD1, SMPD3, IsoPs, IsoFs, NeuroPs, NeuroFs, miRNAs). | 24 months | biomarkers will be measured in pg/ml |
| Variations in echocardiographic parameters of pulmonary hypertension (PH) before and after mesenchymal cell therapy. | 24 months | NO PH (type I less 35%), MILD PH (type I-II between 35-50%) , MODERATE PH (type II between 50-70%) and SEVERE PH ( type II-III, more than 70%) |
| Changes in modified respirator score during therapy and up to week 36 of port-menstrual age | 24 months | 0 - 13 (min- max value). Higher score means worse outcome. |
| Changes in Respiratory Severity Score (RSS) during therapy and up to week 36 of port-menstrual age | 24 months | 0-30 (min- max value). Higher score means worse outcome. |
| Incidence of BPD and PH in very low birth weight babies treated with MSC | 24 months | Status on week 36 of post-menstrual age |
| Need for supplemental O2 at home discharge and during follow-up (Number if patients that need supplemental O2). | 24 months | Number if patients that need supplemental O2 |
| Duration of invasive and non-invasive mechanical ventilation. | 24 months | Duration of invasive and non-invasive mechanical ventilation. |
| Use of postnatal corticosteroids indicated | 24 months | For the treatment or prevention of BPD |
| Respiratory readmission rates. | 24 months | During the first year |
| Bayley Neurodevelopmental Scale at 24 months | 24 months | Evaluation of cognitive developement, languaje developement and motor developement. |
| Date and cause of death. | 24 months | Date and cause of death. |
| Date of hospital discharge and respiratory care at discharge. | 24 months | Date of hospital discharge and respiratory care at discharge. |
| Diagnosis and stage of bronchopulmonary dysplasia on week 36 of post-menstrual age according to Jensen | 24 months | (No BPD/grade 1/grade 2/garde 3) |
Countries
Spain
Contacts
Primary ContactMaría Jesús del Cerro, PhD
Backup ContactMaría Álvarez, MD
Outcome results
None listed