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

NCT ID: NCT04873752

Last Updated: 2025-04-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-06-02
• Study Completion Date: 2024-02-15

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.

Detailed Description

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.

Conditions

Cerebral Palsy; Periventricular Leukomalacia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

UDI-001

Four cycles with 8 administrations

Group Type: EXPERIMENTAL

UDI-001

Intervention Type: BIOLOGICAL

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

Interventions

UDI-001

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 12 Months
• Maximum Eligible Age: 24 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Rohto Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sumito Okawa

Role: STUDY_DIRECTOR

Rohto Pharmaceutical Co., Ltd.

Locations

Osaka City University Hospital

Osaka, Osaka, Japan

Countries

Japan

Other Identifiers

UDI-001-0555

Identifier Type: -

Identifier Source: org_study_id