Efficacy and Safety of Pneumostem® for IVH in Premature Infants (Phase 2a)

Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of IVH in Premature Infants (Phase 2a)

The objective of this study is to evaluate the efficacy and safety of a single intraventricular administration of Pneumostem® for treatment of Intraventricular hemorrhage (IVH) in high-risk premature infants by comparing Pneumostem-treated group with a control group.

Study Overview

• Status: Active, not recruiting
• Conditions: Cell Transplantation
• Intervention / Treatment: Drug: Normal saline; Drug: Pneumostem

• Study Type: Interventional
• Enrollment (Estimated): 22
• Phase: Phase 2

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 06351
    • Samsung Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 4 weeks (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• IVH grade 3-4
• age : within postnatal day 28
• gestational age: 23-<34 weeks

Exclusion Criteria:

• Patient with severe congenital abnormalities
• Patient with antenatal brain hemorrhage
• Patient with asphyxia or Hypoxic ischemic encepalophathy
• Patient with chromosome anomalies (i.e. Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc)
• Patient with a concurrent severe congenital infection (i.e. Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc)
• Patient withCRP > 30 mg/dL; Severe sepsis or shock
• Patient with a history of participating in other clinical studies
• Patient who is considered inappropriate to participate in the study by the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Control; Control group receives placebo medication (normal saline)	Drug: Normal saline; direct intracerebroventricular injection of normal saline via ventricular tap
Experimental: MSC group; MSC group receives mesenchymal stem cells transplantation (Pneumostem)	Drug: Pneumostem; direct intracerebroventricular injection of mesenchymal stem cells via ventricular tap; Other Names: Umbilical cord blood derived mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Shunt or death	Shunt operation or death	40 weeks of corrected age

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Death	Death	at 2years
Developmental delay in gross motor function, 2y	Cerebral palsy, gross motor function classification system score ≥2	at 2years
Developmental delay Bayley -3	Bayley-3 scores <70	at 2years
Developmental delay in KDST, 2y	Korean Developmental Screening Test scores, below two standard deviations was classified as delayed	at 2 years
Language delay	Korean Sequenced Language Scale for Infant score	at 2 years, at 5 years
Developmental delay in KDST, 5y	Korean Developmental Screening Test scores, below two standard deviations was classified as delayed	at 5 years
Developmental delay in gross motor function	Cerebral palsy, gross motor function classification system score ≥2	at 2years
Developmental delay in gross motor function, 5y	Cerebral palsy, gross motor function classification system score ≥2	at 5years
epilepsy	epilepsy required anti-seizure medication or treatment	at 2 years, at 5 years

Collaborators and Investigators

• Sponsor: Samsung Medical Center
• Investigators: Study Director: So Yoon Ahn, MD. Ph.D, Samsung Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): March 28, 2017
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: August 31, 2016
• First Submitted That Met QC Criteria: September 6, 2016
• First Posted (Estimated): September 7, 2016

Study Record Updates

• Last Update Posted (Actual): April 13, 2025
• Last Update Submitted That Met QC Criteria: April 8, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Premature Birth
• Other Study ID Numbers: 2016-06-005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No