Cerebral Palsy, Periventricular Leukomalacia
Conditions
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.
Interventions
BIOLOGICALUDI-001
2.5 x 10\^6 cells/kg of UDI-001 are administered intravenously. One cycle consists of twice a week administrations.
Sponsors
Rohto Pharmaceutical Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Months to 24 Months
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety: Adverse Event | until Week 52 | Adverse events which appear in the participants after the treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Gross Motor Function Measure Score (GMFM Score) | baseline to Week 52 | Difference and change in GMFM score |
| Gross Motor Function Classification System (GMFCS) | baseline to Week 52 | Improvement rate of GMFCS |
| Function Independence Measure for Children (WeeFIM) | baseline to Week 52 | Change in WeeFIM score |
| Kyoto Scale of Psychological Development Test 2001 (KSPD) | baseline to Week 52 | Change in and Improvement rate of KSPD |
Countries
Japan
Outcome results
None listed