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

Phase 1

Active, not recruiting

• Conditions: Cerebral Palsy; Periventricular Leukomalacia
• Interventions: Biological: UDI-001

• Registration Number: NCT04873752
• Lead Sponsor: Rohto Pharmaceutical Co., Ltd.

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.

Study Timeline

• Study First Submitted: 2021-04-30
• Study First Submitted QC: 2021-04-30
• Study First Posted: 2021-05-05
• Study Start: 2021-06-02
• Primary Completion: 2023-07-12
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-14
• Last Update Posted: 2023-11-18
• Study Completion: 2024-03

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 6

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)

Read More

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

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
UDI-001	UDI-001	Four cycles with 8 administrations

Primary Outcome Measures

Name	Time	Method
Safety: Adverse Event	until Week 52	Adverse events which appear in the participants after the treatment

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Osaka City University Hospital; 🇯🇵 Osaka, Japan