PNEUMOSTEM® for Improving Respiratory Outcomes in Very Premature Infants Diagnosed With Early Pulmonary Arterial Hypertension

NCT07368088 | Not Yet Recruiting Phase 1

• 1 other identifier: 2025-10-080
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to evaluate the safety and potential efficacy of PNEUMOSTEM® for improving respiratory outcomes in very premature infants diagnosed with Early Pulmonary Arterial Hypertension. The main questions it aims to answer are:

• In very premature infants diagnosed with early pulmonary arterial hypertension, will a single intratracheal administration of PNEUMOSTEM®(Allogeneic umbilical cord blood-derived mesenchymal stem cells) result in improvement of pulmonary arterial hypertension based on echocardiographic assessment?
• In very premature infants diagnosed with early pulmonary arterial hypertension who show improvement of pulmonary arterial hypertension based on echocardiographic assessment following a single intratracheal administration of PNEUMOSTEM®(Allogeneic umbilical cord blood-derived mesenchymal stem cells), at what time point does this improvement occur? Participants will:
• Single intratracheal dose of PNEUMOSTEM® at 2.0 x 10,000,000 cells/kg
• Acute adverse event monitoring: 24 hours post-administration for safety assessment
• Follow- up time points: Day 1(Baseline, PNEUMOSTEM® administration), Day 2, Week 1, Week 2, Postnatal Day 28, PMA 36\~40 weeks

Conditions

Premature Infants; Pulmonary Arterial Hypertension (PAH)

Outcome Measures

Primary Outcomes (12)

• Time point(week) of complete reversal of cardiac shunt direction

  Time from PNEUMOSTEM administration to complete reversal of intracardiac shunt from right-to-left to complete left-to-right direction, as assessed by echocardiography.

  Biweekly from day after PNEUMOSTEM administration until postnatal day 28

• Time to normalization of ventricular septal configuration

  Time from PNEUMOSTEM administration to resolution of flattened interventricular septum or disappearance of D-shaped left ventricle at end-systole, as assessed by echocardiography.

  Once between postnatal day 28 and PMA 36~40 weeks

• Change in duration of pulmonary hypertension medication use

  Change in duration of pulmonary hypertension medication use by medication type following PNEUMOSTEM administration compared to standard care. Duration will be measured in days for each medication type used to treat pulmonary hypertension.

  Daily on PNEUMOSTEM administration day and the next day

• Change in duration of mechanical ventilation

  Change in total duration of mechalical ventilator use following PNEUMOSTEM andministration compared to standard care. Duration will be measured in days from initiation to discontinuation of mechanical ventilation.

  Weekly at week 1 and week 2 after PNEUMOSTEM administration

• Change in duration of supplemental oxygen therapy.

  Change in total duration of supplemental oxygen use following PNEUMOSTEM administration compared to standard care. Duration will be measured in days from initiation to discontinuation of oxygen therapy.

  Weekly at week 1 and week 2 after PNEUMOSTEM administration

• Incidence of Bronchopulmonary Dysplasia(BPD)

  Inicidence of bronchopulmonary dysplasia assessed according to standard diagnostic criteria(requirement for supplemental oxygen at 28 days of postnatal age and/or at 36 weeks postmenstrual age).

  At postnatal day 28 and at between PMA 36~40 weeks

• Severity of Bronchopulmonary Dysplasia(BPD)

  Severity assessment of bronchopulmonary dysplasia categorized according to the definition of NIHCD 2001, NICHD 2018 and JENSEN 2019.

  At postnatal day 28 and at between PMA 36~40 weeks

• Incidence of Retinopathy of Prematurity(ROP)

  Incidence of retinopathy of prematurity assessed by ophthalmologic examination according to the International Classification of Retinopathy of Prematurity.

  Once at between PMA 36~40 weeks

• Severity of Retinopathy of Prematurity(ROP)

  Severity assessment of retinopathy of prematurity including staging(Stage 1\~5), zone(Zone I, II, or III), and presence of pulse disease. Assessment of treatment requirements including lase photocoagulation, anti-VEGF injection, or surgical intervention.

  Once at between PMA 36~40 weeks

• Time point of brain injury ditection on Brain MRI

  Time point of early detection of brain injury or neurodevelopmental abnormalities on brain MRI performed at postmenstrual age 36\~40 weeks. Brain injuries assessed include intraventricular hemorrhage, periventricular leukomalacia, and other structural abnormalities.

  Once at between PMA 36~40 weeks

• Incidence of Respiratory-related mortality

  Incidence of respiratory-related moratlity confirmed by medical records and information survey. Respiratory-related mortality is defined as death primarily attributed to respiratory failure, pulmonary hypertension, or complications of bronchopulmonary dysplasia.

  From PNEUMOSTEM administration to PMA 36~40 weeks

• Tiem point of respiratory-related mortality

  Postnatal age(in days) at which respiratory-related death occurs in participants who experience this outcome. Time will be measured from PNEUMOSTEM administration to the date of death.

  From PNEUMOSTEM administration to PMA 36~40 weeks

Secondary Outcomes (18)

• Incidence of Adverse Events Assessed by latest version of CTCAE

  From PNEUMOSTEM administration to PMA 36~40 weeks

• Change in anthropometric measurements - Weight

  From PNEUMOSTEM administration to PMA 36~40 weeks

• Change in anthropometric measurements - Head Circumference

  From PNEUMOSTEM administration to PMA 36~40 weeks

• Abnormal findings on physical examination - Skin and Head/Neck

  From PNEUMOSTEM administration to PMA 36~40 weeks

• Abnormal findings on physical examination - Cardiovascular and Respiratory systems

  From PNEUMOSTEM administration to PMA 36~40 weeks

Study Arms (1)

PNEUMOSTEM® treatment arm

EXPERIMENTAL

PNEUMOSTEM® will be administered intratracheally as a single dose of 2.0x10,000,000 cells/kg on Day 1.

Biological: PNEUMOSTEM®

Interventions

PNEUMOSTEM® BIOLOGICAL

PNEUMOSTEM® will be administered intratracheally as a single dose of 2.0x10000000 cells/kg on Day 1.

Also known as: Cell therapy product

PNEUMOSTEM® treatment arm

Eligibility Criteria

• Age: 1 Day - 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Premature infants within 2 weeks of birth with a gestational age of 28 weeks or less or birth weight of less than 1,250g who require continuous invasive mechanical ventilation
• When diagnosed with early pulmonary arterial hypertension satisfying condition ① or ② up to 14 days after birth:
• When on or more of the following abnormal findings are present on echocardiography performed between 4 and 14 days after birth (findings at 1-3 days after birth correspond to early neonatal transition):
• Sytemic or suprasystemic pulmonary artery pressure \>40mmHg(based on peak Doppler velocity of tricuspid regurgitation)
• Right-to-left or bidirectional shunt through patent ductus arteriosus, foramen ovale, or atrial septal defect
• Flattened interventricular septum or D-shaped left ventricle at end systole ② When receiving nitric oxide(NO) inhalation therapy for persistent pulmonary hypertension of the newborn(PPHN) within 3 days after birth

You may not qualify if:

• Those witth cyanotic congenital heart defects or acyanotic congenital heart defects causing heart failure, excluding patent ductus arteriosus in premature infatns
• Those with severe pulmonary malformations such as congenital diaphragmatic hernia or congenital cystic lung disease
• Those who underwent surgery within 72 hours before or after administration of the investigational cell product, or those for whom surgery is anticipated
• Those who received surfactant within 24 hours prior to administration of the investigattional cell product
• Those with chromosomal abnormalities accompanied by severe malformations(such as Edwards syndrome, Patau syndrome, Down syndrome, etc.) and severe congenital malformations(such as hydrocephalus, encephalocele, etc.)
• Those with severe congenital infectious diseases(such as herpes, toxoplasmosis, rubella, syphilis, AIDS, etc.)
• Those with severe sepsis or shock due to active infection not adequately treated with antibiotics
• Those who have a history of participation in other advanced regenerative medicine clinical studies or clinical trials
• Others deemed inappropriate by tthe investigator to participate in this advanced regenerative medicine clinical study

Sponsors & Collaborators

• Samsung Medical Center: lead

Study Sites (1)

Samsung Medical Center, 81, Irwon-ro, Gangnam-gu, Seoul, Republic of Korea

Seoul, 06351, South Korea

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Premature Birth

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases; Obstetric Labor, Premature; Obstetric Labor Complications; Pregnancy Complications; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases

Central Study Contacts

So Yoon Ahn, M.D, Ph.D

CONTACT

+82-10-4038-0460; yoon.ahn.neo@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Model Details: Single center, Single-arm study

Study Record Dates

• First Submitted: November 25, 2025
• First Posted: January 26, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: January 26, 2026

Record last verified: 2025-12

Locations

KR (1)