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NCT Number: NCT04873752

A 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.

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Key information

Conditions

Cerebral Palsy Brain Damage, Chronic Brain Diseases Cardiovascular Diseases Central Nervous System Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Encephalomalacia Infant, Newborn, Diseases Infant, Premature, Diseases Leukomalacia, Periventricular Nervous System Diseases Periventricular Leukomalacia Vascular Diseases

Age range

12 month–24 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Osaka City University Hospital

Osaka, 545-8586, Japan

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

UDI-001

Biological

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

Primary outcomes

  1. Safety: Adverse Event

    Time frame: until Week 52

    Adverse events which appear in the participants after the treatment

Secondary outcomes

  1. Gross Motor Function Measure Score (GMFM Score)

    Time frame: baseline to Week 52

    Difference and change in GMFM score

  2. Gross Motor Function Classification System (GMFCS)

    Time frame: baseline to Week 52

    Improvement rate of GMFCS

  3. Function Independence Measure for Children (WeeFIM)

    Time frame: baseline to Week 52

    Change in WeeFIM score

  4. Kyoto Scale of Psychological Development Test 2001 (KSPD)

    Time frame: baseline to Week 52

    Change in and Improvement rate of KSPD

Sponsors and collaborators

Lead sponsor

Rohto Pharmaceutical Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2023
Study completion
2024
First posted
May 5, 2021
Registry last updated
Apr 6, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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