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

Официальное название

A Clinical Study of Advanced Regenerative Medicine to Evaluate the Safety and Potential Efficacy of PNEUMOSTEM® for Improving Respiratory Outcomes in Very Premature Infants Diagnosed With Early Pulmonary Arterial Hypertension

Обзор

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

Вмешательства

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

Первичные конечные точки

• Time point(week) of complete reversal of cardiac shunt direction [Срок оценки: Biweekly from day after PNEUMOSTEM administration until postnatal day 28]
• Time to normalization of ventricular septal configuration [Срок оценки: Once between postnatal day 28 and PMA 36~40 weeks]
• Change in duration of pulmonary hypertension medication use [Срок оценки: Daily on PNEUMOSTEM administration day and the next day]
• Change in duration of mechanical ventilation [Срок оценки: Weekly at week 1 and week 2 after PNEUMOSTEM administration]
• Change in duration of supplemental oxygen therapy. [Срок оценки: Weekly at week 1 and week 2 after PNEUMOSTEM administration]
• Incidence of Bronchopulmonary Dysplasia(BPD) [Срок оценки: At postnatal day 28 and at between PMA 36~40 weeks]
• Severity of Bronchopulmonary Dysplasia(BPD) [Срок оценки: At postnatal day 28 and at between PMA 36~40 weeks]
• Incidence of Retinopathy of Prematurity(ROP) [Срок оценки: Once at between PMA 36~40 weeks]
• Severity of Retinopathy of Prematurity(ROP) [Срок оценки: Once at between PMA 36~40 weeks]
• Time point of brain injury ditection on Brain MRI [Срок оценки: Once at between PMA 36~40 weeks]

Вторичные конечные точки (12)

• Incidence of Adverse Events Assessed by latest version of CTCAE [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in anthropometric measurements - Weight [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in anthropometric measurements - Head Circumference [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Abnormal findings on physical examination - Skin and Head/Neck [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Abnormal findings on physical examination - Cardiovascular and Respiratory systems [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Abnormal findings on physical examination - Abdomen and Genitalia [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Abnormal findings on physical examination - Nervous and Musculoskeletal systems [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in vital signs - Blood pressure [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in vital signs - Body temperature [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in vital signs - Heart rate [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in vital signs - Respiratory rate [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]
• Change in vital signs - Oxygen saturation [Срок оценки: From PNEUMOSTEM administration to PMA 36~40 weeks]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

South Korea · 1 центр

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

Идентификаторы

NCT: NCT07368088 · 2025-10-080

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗