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

November 14, 2023 updated by: Rohto Pharmaceutical Co., Ltd.

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

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Osaka, Japan, 545-8586
        • Osaka City University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 2 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety: Adverse Event
Time Frame: until Week 52
Adverse events which appear in the participants after the treatment
until Week 52

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rohto Pharmaceutical Co., Ltd.

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 2, 2021

Primary Completion (Actual)

July 12, 2023

Study Completion (Estimated)

March 1, 2024

Study Registration Dates

First Submitted

April 30, 2021

First Submitted That Met QC Criteria

April 30, 2021

First Posted (Actual)

May 5, 2021

Study Record Updates

Last Update Posted (Estimated)

November 16, 2023

Last Update Submitted That Met QC Criteria

November 14, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UDI-001-0555

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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