The Protocol of Estimation of the Safety and Efficiency of the Method of Transfusion of UCB Hemopoietic Cells to the Patients With Diagnosed Infantile Cerebral Palsy Depending on the Degrees of Compatibility of Donor and Recipient
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- Enrollment
- 150
- Locations
- 2
- Primary Endpoint
- Changes in Ashworth scale score for all child.
Study Overview
Brief Summary
Cerebral palsy is a disorder of movement and posture resulted from a non-progressive lesion or injury of the immature brain. It is a leading cause of childhood-onset disability.
Many experimental animal studies have revealed that umbilical cord blood is useful to repair neurological injury in the brain.
Based on many experimental studies, umbilical cord blood is suggested as a potential therapy for cerebral palsy.
This protocol was developed based on the results of the previously approved protocol of the center NCT03826498 (Allogeneic cord blood transfusion in patients with infantile cerebral palsy), which showed high efficiency in the rehabilitation of patients. The present protocol is intended for revealing the dependence of the clinical effect on the degree of tissue compatibility of umbilical cord blood samples and the recipient
Detailed Description
Cerebral palsy (CP) is a group of neurodevelopmental conditions with abnormal movement and posture resulted from a non-progressive cerebral disturbance. It is the most common cause of motor disability in childhood. Most therapies are palliative rather than restorative. Umbilical cord blood (UCB) may be used as restorative approach for children with CP.
Many experimental animal studies have revealed that UCB is beneficial to improve and repair neurological injuries, this effect achieved due to immune regulation and angiogenesis as well as the neuroprotective effect.
Based on animal studies and some clinical trials, UCB is suggested as a potential therapy for children with CP.
This study is prospective, non randomized (open label) with control group
СLINICAL PURPOSES Estimation of the efficiency of the method of transfusion of umbilical blood hematopoietic cells to patients with diagnosed infantile cerebral palsy depending on the degrees of compatibility of donor and recipient.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 1 Year to 12 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Patient selection criteria (indications for this type of treatment):
- •Patient age from 1 to 12 years;
- •Diagnosis: cerebral palsy, including postnatal damage after ischemic or hemorrhagic strokes, hypoxic or ischemic encephalopathy, periventricular leucomalacia;
- •The presence of I - V lesion levels on the GMFCS - ER (CanChild) scale;
- •The presence of a compatible allogeneic sample suitable for infusion;
- •Parental consent (official guardians)
Exclusion Criteria
- •(contraindications for this type of treatment):
- •Patient age up to 1 year, older than 12 years;
- •The presence of the following diseases in history: heart failure in the stage of decompensation, anemia and other blood diseases;
- •Decompensation of chronic and endocrinological diseases;
- •Acute viral and bacterial infections during the acute clinical phase of the disease;
- •HIV infection, hepatitis of B and C types;
- •Oncological diseases, chemotherapy in the anamnesis;
- •Tuberculosis;
- •Confirmed genetic disorders;
- •A severe form of intellectual disability as a concomitant disease (diagnosis can be ignored, according to the decision of the Medical Committee of the Center);
- •Epileptic seizures with or without medication in the last 6 months before inclusion in the protocol.
Outcomes
Primary Outcomes
Changes in Ashworth scale score for all child.
Time Frame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
The Ashworth scale (AS) measures resistance during passive soft-tissue stretching and is used as a simple measure of spasticity scoring, where: 0. No increase in tone; 1. A slight increase in tone giving catch when the limb is moved in flexion and extension; 2. A more marked increase in tone, but the limb is easily flexed; 3. Considerable increases in tone, passive movement difficult; 4. Limb rigid in flexion or extension. This test will be acquired for the child above 3yrs.
Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.
Time Frame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of sub-scales; lying \& rolling, sitting, crawling \& kneeling, standing, walking, running \& jumping (range: 0\~100, a Higher value means better gross motor function). We reported changes in GMFM between each assessment time point. Categories of outcome table are baseline and values of just subtracting the latter raw scores from the former ones. This test will be acquired for all children.
Gross Motor Function Classification System (GMFCS - ER) scale severity change
Time Frame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
Evaluation of the overall dynamics of treatment. GMFCS is a 5 level clinical classification system that describes the gross motor function of people with cerebral palsy on the basis of self-initiated movement abilities. Distinctions between levels are based on functional abilities; the need for walkers, crutches, wheelchairs, or canes/walking sticks; and to a much lesser extent, the actual quality of movement: Level I: Walks without Limitations Level II: Walks with Limitations Level III: Walks Using a Hand-Held Mobility Device Level IV: Self-Mobility with Limitations; May Use Powered Mobility Level V: Transported in a Manual Wheelchair The Evaluation of the results is carried out by comparing the primary and subsequent indications. Information will be collected from parents.
Number of participants with non-serious and serious adverse events
Time Frame: 1 year
Safety assessment such as adverse events will be registered. Adverse events will be monitored during all trial.
Changes in The Infant Toddler Quality of Life Questionnaire for child above 3yrs.
Time Frame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
ITQOL - the 47-item short-form (ITQOL-SF47) developed for use in infants and toddlers from 2-months-to-5 years of age. Form scores physical, mental and social well being/ For each concept, item responses are scored, summed, and transformed on a scale from 0 (worst health) to 100 (best health). Changes in the completed questionnaire will be assessed. This test will be acquired for the child above 3yrs.
Secondary Outcomes
- Number of Participants with Chimerism (longevity) of infused cell(Baseline, 6 month after first infusion)
- The correlation between the concentration of CD34+ cells in the UCB and the treatment effectiveness degree(1, 6 months (At each infusion))
- The correlation between the concentration of CD14+ cells in the sample of UCB and the treatment effectiveness degree.(1, 6 months (At each infusion))
Investigators
Volchkov Stanislav
Deputy director, Quality assurance director
State-Financed Health Facility "Samara Regional Medical Center Dinasty"