Cerebral Palsy (CP)
Conditions
Keywords
Cerebral palsy, clinical trial, stromal cell, intrathecal, outcome, adverse event, safety
Brief summary
This is a single-center, open-label, single-arm Phase I pilot study designed to evaluate the feasibility and short-term safety of a single intrathecal administration of allogeneic umbilical cord tissue-derived mesenchymal stromal cells (UCT-MSCs) in children with cerebral palsy (CP).
Detailed description
Seven children aged 2-5 years with Gross Motor Function Classification System (GMFCS) levels II-V will receive a single intrathecal dose of allogeneic UCT-MSCs in addition to standard multidisciplinary care. The primary outcomes are procedural feasibility and short-term safety, including the occurrence of adverse events during the procedure, hospitalization, and throughout a 6-month follow-up period. Exploratory outcomes include changes in functional classification and spasticity. This pilot study is intended to provide preliminary safety and feasibility data to support the design of future controlled clinical trials and is not powered to evaluate treatment efficacy.
Interventions
BIOLOGICALallogeneic umbilical cord tissue-derived mesenchymal stromal cells (UCT-MSCs)
A single intrathecal dose of allogeneic umbilical cord tissue-derived mesenchymal stromal cells (UCT-MSCs) at 2 × 10⁶ viable cells/kg body weight
Sponsors
Gadjah Mada University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 5 Years
Healthy volunteers
No
Inclusion criteria
* Children aged 2-5 years. Diagnosis of cerebral palsy, independently confirmed by two experienced pediatric neurologists. Gross Motor Function Classification System (GMFCS) level II-V. Parent(s) or legal guardian(s) able and willing to provide written informed consent.
Exclusion criteria
* History of congenital infection, including toxoplasmosis, rubella, cytomegalovirus, herpes simplex virus, or other clinically relevant congenital infections. Normal findings on brain neuroimaging. Known malignancy. Confirmed genetic or congenital disorder unrelated to cerebral palsy. Advanced systemic disease, including renal insufficiency, hepatic dysfunction, or severe anemia. Previous treatment with stem cell or stromal cell therapy. Participation in another interventional clinical study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Successfully Completed Intrathecal Administration of the Intended UCT-MSC Dose Without Technical Failure and Completed the 6-Month Follow-up | From the intrathecal administration through 6 months after intervention. | Feasibility was defined as successful delivery of the intended dose of allogeneic umbilical cord-derived mesenchymal stromal cells (UCT-MSCs), completion of the intrathecal administration without technical failure, completion of the planned post-procedural observation, participant retention through the 6-month follow-up, and absence of protocol deviations preventing completion of the intervention or follow-up. |
| Number of Participants With Treatment-Emergent Adverse Events Following Intrathecal Administration of Allogeneic Umbilical Cord-Derived Mesenchymal Stromal Cells | From the intrathecal administration through 6 months after intervention. | Safety was assessed by recording immediate and delayed treatment-emergent adverse events occurring during the procedure, during hospitalization, and throughout the 6-month follow-up period. Events of interest included fever, vomiting, headache, irritability, back pain, local procedural complications, infection, allergic or infusion-related reactions, seizure, altered consciousness, neurological deterioration, prolonged hospitalization, and serious adverse events. For each adverse event, the time of onset, maximum severity, duration, management, clinical outcome, seriousness, and investigator-assessed relationship to the intervention were documented. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Gross Motor Function Classification System (GMFCS) Level | Baseline, Month 1, Month 3, and Month 6 | Gross motor function was classified using the Gross Motor Function Classification System (GMFCS), a five-level ordinal classification system ranging from Level I (least severe limitation) to Level V (most severe limitation). Changes in GMFCS level were summarized descriptively and were not interpreted as evidence of treatment efficacy. |
| Manual Ability Classification System (MACS) Level | Baseline, Month 1, Month 3, and Month 6 | Manual ability was classified using the Manual Ability Classification System (MACS), a five-level ordinal classification system ranging from Level I to Level V. Changes in MACS level were summarized descriptively and were not interpreted as evidence of treatment efficacy. |
| Communication Function Classification System (CFCS) Level | Baseline, Month 1, Month 3, and Month 6 | Communication performance was assessed using the Communication Function Classification System (CFCS), a five-level ordinal classification system describing everyday communication performance. Results were summarized descriptively and were not interpreted as evidence of treatment efficacy. |
| Eating and Drinking Ability Classification System (EDACS) Level | Baseline, Month 1, Month 3, and Month 6 | Eating and drinking ability was classified using the Eating and Drinking Ability Classification System (EDACS), a five-level ordinal classification system. Changes in EDACS level were summarized descriptively and were not interpreted as evidence of treatment efficacy. |
| Modified Ashworth Scale (MAS) Score | Baseline, Month 1, Month 3, and Month 6 | Spasticity was assessed using the Modified Ashworth Scale (MAS), an ordinal scale ranging from 0 (no increase in muscle tone) to 4 (affected part rigid in flexion or extension). Muscle groups evaluated were assessed according to the study protocol. MAS scores were summarized descriptively because the scale is ordinal and may be influenced by positioning, participant cooperation, and assessor judgment. |
Countries
Indonesia
Outcome results
None listed