Cerebral Palsy Hypoxic-Ischemic Encephalopathy Intracranial Hemorrhage
Conditions
Keywords
Bone Marrow Mononuclear Cells Autologous Transplantation Intrathecal Injection Rehabilitation Pediatric Brain Injury
Brief summary
This study evaluates the safety and preliminary efficacy of autologous bone marrow mononuclear cell transplantation combined with rehabilitation in children with neurological sequelae caused by hypoxic-ischemic brain injury or intracranial hemorrhage. Participants in the intervention group will receive autologous bone marrow aspiration, mononuclear cell transplantation via intrathecal route, and rehabilitation, while the control group will receive rehabilitation alone. Outcomes will be compared between groups over follow-up time points.
Detailed description
Neurological sequelae following hypoxic-ischemic brain injury and intracranial hemorrhage in children remain a major clinical challenge with limited effective treatment options. Rehabilitation is the current standard of care but often results in incomplete recovery. Autologous bone marrow mononuclear cell transplantation has emerged as a promising therapeutic approach due to its potential neuroregenerative and neuroprotective effects. This phase II study aims to evaluate the safety and preliminary efficacy of combining autologous bone marrow mononuclear cell transplantation with rehabilitation compared to rehabilitation alone. The study will also contribute to the development of a standardized technical protocol for this intervention.
Interventions
Autologous bone marrow mononuclear cells (BMMNCs) are collected from the patient's iliac crest under sterile conditions. Bone marrow aspiration is performed under appropriate anesthesia, followed by processing to isolate mononuclear cells using density gradient centrifugation. The prepared BMMNCs are administered via intrathecal injection under aseptic conditions. The dosage of cells is determined based on body weight and viability criteria. Patients are monitored for immediate and delayed adverse events following transplantation. In addition to cell transplantation, participants receive standardized rehabilitation therapy, including physical therapy, occupational therapy, and neurodevelopmental interventions, according to institutional protocols. This combined intervention aims to enhance neurological recovery through both regenerative cellular therapy and functional rehabilitation.
Sponsors
National Children's Hospital, Vietnam
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
* Children diagnosed with cerebral palsy * Age from 12 months to 72 months * Cerebral palsy caused by hypoxic-ischemic brain injury or intracranial hemorrhage * Gross Motor Function Classification System (GMFCS) level III to V * Completed required laboratory tests according to the study protocol * Written informed consent obtained from the child's parent or legal guardian
Exclusion criteria
* Children with acute infection at the time of screening or enrollment * Children with severe coagulopathy or contraindications to bone marrow aspiration * Children with severe systemic diseases (e.g., severe cardiac, hepatic, or renal failure) * Children with contraindications to anesthesia or intrathecal transplantation procedures * Children with a history of malignancy * Children who are unable to participate in or complete the rehabilitation program * Parents or legal guardians who do not provide informed consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Gross Motor Function Measure (GMFM-88) total score from baseline to 12 months | Baseline and 12 months | Gross motor function will be assessed using the Gross Motor Function Measure (GMFM-88), a validated and standardized clinical scale for evaluating motor function in children with neurological impairment. The primary outcome is the change in GMFM-88 total score from baseline to 12 months. The change in score will be calculated and compared between the intervention group (autologous bone marrow mononuclear cell transplantation plus rehabilitation) and the control group (rehabilitation alone). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in GMFM score at 3, 6, 9 months | Baseline and 3, 6, 9 months | Gross motor function will be assessed using the Gross Motor Function Measure (GMFM-88), a standardized clinical tool for children with cerebral palsy and related neurological impairment. The change in GMFM-88 total score from baseline to 3, 6,9 months will be calculated and compared between the intervention and control groups. |
| Change in Gross Motor Function Classification System (GMFCS) level from baseline to 12 months | Baseline and 12 months | Gross motor function classification will be assessed using the Gross Motor Function Classification System (GMFCS), which categorizes motor function based on self-initiated movement abilities, particularly sitting and mobility. Changes in GMFCS level from baseline to 12 months will be evaluated and compared between the intervention and control groups. |
| Change in Manual Ability Classification System (MACS or Mini-MACS) level from baseline to 12 months | Baseline and 12 months | Manual ability will be assessed using the Manual Ability Classification System (MACS) or Mini-MACS depending on the child's age. Changes in manual ability level from baseline to 12 months will be evaluated and compared between the intervention and control groups. |
| Incidence of transplantation-related adverse events | From intervention through 12 months follow-up | Adverse events related to bone marrow aspiration, anesthesia, intrathecal transplantation, and follow-up will be recorded. These include local pain at the aspiration site, low back pain after transplantation, fever, irritability, vomiting, rash, and other procedure-related complications. The incidence and severity of adverse events will be monitored and summarized throughout the study. |
Countries
Vietnam
Contacts
CONTACTDAT HUU TRAN
Outcome results
None listed