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

Cerebral Palsy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04873752
• Other study ID #: UDI-001-0555
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: June 2, 2021
• Est. completion date: March 2024

Study information

• Verified date: November 2023
• Source: Rohto Pharmaceutical Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 6
• Est. completion date: March 2024
• Est. primary completion date: July 12, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 24 Months

Eligibility

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

Related Conditions & MeSH terms

• Cerebral Palsy
• Leukomalacia, Periventricular
• Periventricular Leukomalacia

Intervention

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

Locations

Country	Name	City	State
Japan	Osaka City University Hospital	Osaka

Sponsors (1)

Lead Sponsor	Collaborator
Rohto Pharmaceutical Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Adverse Event	Adverse events which appear in the participants after the treatment	until Week 52
Secondary	Gross Motor Function Measure Score (GMFM Score)	Difference and change in GMFM score	baseline to Week 52
Secondary	Gross Motor Function Classification System (GMFCS)	Improvement rate of GMFCS	baseline to Week 52
Secondary	Function Independence Measure for Children (WeeFIM)	Change in WeeFIM score	baseline to Week 52
Secondary	Kyoto Scale of Psychological Development Test 2001 (KSPD)	Change in and Improvement rate of KSPD	baseline to Week 52