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

ROHTO Pharmaceutical

Status and phase

Completed

Phase 2

Phase 1

Conditions

Cerebral Palsy

Periventricular Leukomalacia

Treatments

Biological: UDI-001

Study type

Interventional

Funder types

Industry

Identifiers

NCT04873752

UDI-001-0555

Details and patient eligibility

About

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

Full 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.

Enrollment

6 patients

Sex

All

Ages

12 to 24 months old

Volunteers

No 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