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

NCT04873752 | Completed Phase 1

• 1 other identifier: UDI-001-0555
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Cerebral Palsy; Periventricular Leukomalacia

Outcome Measures

Primary Outcomes (1)

• Safety: Adverse Event

  Adverse events which appear in the participants after the treatment

  until Week 52

Secondary Outcomes (4)

• Gross Motor Function Measure Score (GMFM Score)

  baseline to Week 52

• Gross Motor Function Classification System (GMFCS)

  baseline to Week 52

• Function Independence Measure for Children (WeeFIM)

  baseline to Week 52

• Kyoto Scale of Psychological Development Test 2001 (KSPD)

  baseline to Week 52

Study Arms (1)

UDI-001

EXPERIMENTAL

Four cycles with 8 administrations

Biological: UDI-001

Interventions

UDI-001 BIOLOGICAL

2.5 x 10\^6 cells/kg of UDI-001 are administered intravenously. One cycle consists of twice a week administrations.

UDI-001

Eligibility Criteria

• Age: 12 Months - 24 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Rohto Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Osaka City University Hospital

Osaka, Osaka, 545-8586, Japan

Location

MeSH Terms

Conditions

Cerebral Palsy; Leukomalacia, Periventricular

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebrovascular Disorders; Encephalomalacia; Vascular Diseases; Cardiovascular Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Sumito Okawa

  Rohto Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2021
• First Posted: May 5, 2021
• Study Start: June 2, 2021
• Primary Completion: July 12, 2023
• Study Completion: February 15, 2024
• Last Updated: April 6, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)