Mesenchymal Stem Cell Therapy for Bronchopulmonary Dysplasia in Preterm Babies

• ClinicalTrials.gov Identifier: NCT02443961
• Recruitment Status: Active, not recruiting
• First Posted: May 14, 2015
• Last Update Posted: February 3, 2021
• 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
• Information provided by (Responsible Party): Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal

Tracking Information

• First Submitted Date: May 4, 2015
• First Posted Date: May 14, 2015
• Last Update Posted Date: February 3, 2021
• Actual Study Start Date: April 2, 2019
• Actual Primary Completion Date: April 2, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 11, 2015)

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) [ Time Frame: 24 months ]

Number of participants with adverse events as a measure of safety and tolerability

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 11, 2015)

• 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. [ Time Frame: 24 months ]
  biomarkers will be measured in pg/ml
• Changes in the echocardiographic parameters related with PH and preterm birth, in patients treated with MSC (Number of participants with echocardiographic adverse events) [ Time Frame: 24 months ]
  Flattening of the interventricular septum will be the main parameter (tipe I, I-II, II, II-III OR III)
• Incidence of BPD and PH in very low birth weight babies treated with MSC [ Time Frame: 24 months ]
  Diagnosed at 36 weeks of postmenstrual age

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Mesenchymal Stem Cell Therapy for Bronchopulmonary Dysplasia in Preterm Babies
• Official Title: Clinical Trial: Security and Feasibility of Mesenchymal Stem Cell Therapy in Treatment and Prevention of Bronchopulmonary Dysplasia in Preterm Babies
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Other
• Condition: Bronchopulmonary Dysplasia

Intervention

Biological: Mesenchymal Stem Cell (MSC) therapy

3 doses of 5 million MSC will be administered

Study Arms

Experimental: Mesenchymal Stem Cell (MSC) therapy

There will only be one treatment arm to evaluate the security of the treatment with MSC.

Intervention: Biological: Mesenchymal Stem Cell (MSC) therapy

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: May 11, 2015): 10
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: April 2025
• Actual Primary Completion Date: April 2, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 1 Month to 28 Weeks (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Spain

Administrative Information

• NCT Number: NCT02443961
• Other Study ID Numbers: PULMESCEL-1
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Responsible Party: Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
• Study 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

• PRS Account: Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
• Verification Date: January 2021