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

Sponsor

Rohto Pharmaceutical Co., Ltd. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04873752

Collaborator

(none)

Enrollment

Location

Arm

24.9

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cerebral Palsy Periventricular Leukomalacia	Biological: UDI-001	Phase 1/Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

6 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jun 2, 2021

Anticipated Primary Completion Date :

Mar 1, 2023

Anticipated Study Completion Date :

Jul 1, 2023

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: UDI-001

Four cycles with 8 administrations

Biological: UDI-001

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

Outcome Measures

Primary Outcome Measures

Safety: Adverse Event [until Week 52]
Adverse events which appear in the participants after the treatment

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Months to 24 Months

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Osaka City University Hospital	Osaka		Japan	545-8586

Sponsors and Collaborators

Rohto Pharmaceutical Co., Ltd.

Investigators

Study Director: Sumito Okawa, Rohto Pharmaceutical Co., Ltd.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Rohto Pharmaceutical Co., Ltd.

ClinicalTrials.gov Identifier:

NCT04873752

Other Study ID Numbers:

UDI-001-0555

First Posted:

May 5, 2021

Last Update Posted:

Sep 16, 2021

Last Verified:

Sep 1, 2021

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Cerebral Palsy

Leukomalacia, Periventricular

Study Results

No Results Posted as of Sep 16, 2021