A Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy

Study Overview
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Brief Summary
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UDI-001 is administered to pediatric patients with cerebral palsy attributed to periventricular leukomalacia (PVL) multiple times to investigate its safety and efficacy.

Detailed Description
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This is the first study of UDI-001 to be administered to children. Pediatric patients with cerebral palsy attributed to PVL are enrolled to the study and umbilical cord derived mesenchymal stromal cells (UC-MSCs) are administered to those patients multiple times intravenously. Safety and efficacy of UC-MSCs are evaluated for 52 weeks after the first administration.

Official Title
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An Exploratory Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy

Conditions
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Cerebral Palsy, Periventricular Leukomalacia

Intervention / Treatment
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* Biological: UDI-001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age >= 12 months and < 24 months corrected age at the time of informed consent Diagnosed with cerebral palsy Diagnosed with PVL GMFCS level between II and IV Able to obtain written informed consent from parents (legal representative) Exclusion Criteria: Presence of progressive neurological disease Presence of congenital anomaly Diagnosed with Grade 3 or more severe intraventricular hemorrhage Body weight < 5kg Profound intellectual disorder Complication of serious infection such as sepsis Requirement of mechanical ventilation Complication of serious organ failure such as kidney, liver or heart diseases or others and considered to be inappropriate Diagnosed with or suspected of hypsarrhythmia Positive for HBV, HCV, HIV or HTLV-1 Patients who have received cell therapy Patients who have undergone selective dorsal rhizotomy or received botulinum toxin products within a certain period of time

Ages Eligible for Study
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Minimum Age: 12 Months
Maximum Age: 24 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: UDI-001<br>Four cycles with 8 administrations | Biological: UDI-001<br>* 2.5 x 10^6 cells/kg of UDI-001 are administered intravenously. One cycle consists of twice a week administrations.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety: Adverse Event | Adverse events which appear in the participants after the treatment | until Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gross Motor Function Measure Score (GMFM Score) | Difference and change in GMFM score | baseline to Week 52 | 
| Gross Motor Function Classification System (GMFCS) | Improvement rate of GMFCS | baseline to Week 52 | 
| Function Independence Measure for Children (WeeFIM) | Change in WeeFIM score | baseline to Week 52 | 
| Kyoto Scale of Psychological Development Test 2001 (KSPD) | Change in and Improvement rate of KSPD | baseline to Week 52 |