Mesenchymal Stem Cell Therapy for Bronchopulmonary Dysplasia in Preterm Babies

Clinical Trial: Security and Feasibility of Mesenchymal Stem Cell Therapy in Treatment and Prevention of Bronchopulmonary Dysplasia in Preterm Babies

Bronchopulmonary Dysplasia (BPD) is the most frequent disease related to a premature birth, 15-50% of very low birth newborns (<1500 gr.) will develop BPD. The prevalence of BPD is increasing due to the advances in neonatology, with a rise in the survival of smaller and more premature babies. The etiology of BPD is multifactorial, in which oxygen, maternal chorioamnionitis, insufficient pulmonary maturation etc. have an important role. These factors lead to a pathological development of the lung and pulmonary vessels, developing secondary Pulmonary Hypertension (PH). Nowadays there is no efficient treatment; this generates a important sanitary burden and a decrease in life quality. Multiple experimental models in mice have studied Mesenchymal Stem Cell (MSC) therapy as prevention of BPD, also recently some clinical trials have tried this therapy on premature newborns with promising results. Hypothesis: MSC therapy in patients at high risk of BPD prevents pulmonary lesions. Methods: The investigators have designed a clinical trial to evaluate the feasibility and security of MSC therapy in patients at high risk of developing BPD.

Study Overview

• Status: Completed
• Conditions: Bronchopulmonary Dysplasia
• Intervention / Treatment: Biological: Mesenchymal Stem Cell (MSC) therapy

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• Spain
  • A Coruña, Spain
    • Hospital Universitario A Coruña
  • Madrid, Spain
    • Hospital Clinico San Carlos
  • Madrid, Spain
    • Hospital Universitario La Paz
  • Valencia, Spain
    • Hospital Universitario y Politecnico La Fe

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 6 months (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Preterm newborns ≤ 28 weeks gestational age
• Birth Weight <1250 gr.
• Still on of mechanical ventilation FiO2 > 0,3 at day + 14

Exclusion Criteria:

• Other congenital pathology (pulmonary malformations, active pulmonary bleeding, renal malformations, CHD, malformative syndromes, chromosomopathies)
• Severe neurological lesion.
• HIV infection
• Cardiovascular instability due to any cause
• 72 hours after mayor surgery
• Necrotizing enterocolitis grades II or higher, according to Bell classification, at the time of inclusion.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mesenchymal Stem Cell (MSC) therapy; There will only be one treatment arm to evaluate the security of the treatment with MSC.	Biological: Mesenchymal Stem Cell (MSC) therapy; 3 doses of 5 million MSC will be administered

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility and security of MSC therapy in very low birth weight preterm babies at risk of developing bronchopulmonary dysplasia (Number of participants with adverse events)	Number of participants with adverse events as a measure of safety and tolerability	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Biomarker analysis (IL-1beta, IL-6, IP-10, INF-gamma, TGF beta, NLRP3, RAGE, HMGB1, VEGFA, GREMLIN1, sVEGFR1, IGF, ENDOTHELIN-1, SMPD-1, SP-D, SMPD3.	biomarkers will be measured in pg/ml	24 months
Changes in the echocardiographic parameters related with PH and preterm birth, in patients treated with MSC (Number of participants with echocardiographic adverse events)	Flattening of the interventricular septum will be the main parameter (tipe I, I-II, II, II-III OR III)	24 months
Incidence of BPD and PH in very low birth weight babies treated with MSC	Diagnosed at 36 weeks of postmenstrual age	24 months

Collaborators and Investigators

• Sponsor: Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
• Collaborators: Instituto de Salud Carlos III; Fundación de Ayuda a la Investigación sobre la Hipertensión pulmonar
• Investigators: Principal Investigator: Maria Jesus del Cerro, PhD, IRYCIS. Hospital Universitario Ramón y Cajal. Madrid. Spain

Study record dates

Study Major Dates

• Study Start (Actual): April 2, 2019
• Primary Completion (Actual): April 2, 2020
• Study Completion (Actual): July 7, 2022

Study Registration Dates

• First Submitted: May 4, 2015
• First Submitted That Met QC Criteria: May 11, 2015
• First Posted (Estimate): May 14, 2015

Study Record Updates

• Last Update Posted (Actual): March 30, 2023
• Last Update Submitted That Met QC Criteria: March 29, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: pulmonary hypertension; mesenchymal stem cells; Bronchopulmonary dysplasia; stem cell therapy; very low weight preterm babies
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Lung Injury; Infant, Premature, Diseases; Ventilator-Induced Lung Injury; Hyperplasia; Bronchopulmonary Dysplasia
• Other Study ID Numbers: PULMESCEL-1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No