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

Primary Purpose

Cerebral Palsy, Periventricular Leukomalacia

Status
Active
Phase
Phase 1
Locations
Japan
Study Type
Interventional
Intervention
UDI-001
Sponsored by
Rohto Pharmaceutical Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Palsy

Eligibility Criteria

12 Months - 24 Months (Child)All SexesDoes not accept 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

Sites / Locations

  • Osaka City University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

UDI-001

Arm Description

Four cycles with 8 administrations

Outcomes

Primary Outcome Measures

Safety: Adverse Event
Adverse events which appear in the participants after the treatment

Secondary Outcome Measures

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

Full Information

First Posted
April 30, 2021
Last Updated
October 25, 2022
Sponsor
Rohto Pharmaceutical Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT04873752
Brief Title
A Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy
Official Title
An Exploratory Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 2, 2021 (Actual)
Primary Completion Date
March 2023 (Anticipated)
Study Completion Date
July 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Rohto Pharmaceutical Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy, Periventricular Leukomalacia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
UDI-001
Arm Type
Experimental
Arm Description
Four cycles with 8 administrations
Intervention Type
Biological
Intervention Name(s)
UDI-001
Intervention Description
2.5 x 10^6 cells/kg of UDI-001 are administered intravenously. One cycle consists of twice a week administrations.
Primary Outcome Measure Information:
Title
Safety: Adverse Event
Description
Adverse events which appear in the participants after the treatment
Time Frame
until Week 52
Secondary Outcome Measure Information:
Title
Gross Motor Function Measure Score (GMFM Score)
Description
Difference and change in GMFM score
Time Frame
baseline to Week 52
Title
Gross Motor Function Classification System (GMFCS)
Description
Improvement rate of GMFCS
Time Frame
baseline to Week 52
Title
Function Independence Measure for Children (WeeFIM)
Description
Change in WeeFIM score
Time Frame
baseline to Week 52
Title
Kyoto Scale of Psychological Development Test 2001 (KSPD)
Description
Change in and Improvement rate of KSPD
Time Frame
baseline to Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
24 Months
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sumito Okawa
Organizational Affiliation
Rohto Pharmaceutical Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Osaka City University Hospital
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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