Clinical trial NCT04098029: Estimation of the Safety and Efficiency Transfusion of HLA Matched CBU in Patients With CP
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Cerebral Palsy
Keywords: Cerebral palsy
CP
Cord Blood
Stem cells
HLA
Lead sponsor: State-Financed Health Facility "Samara Regional Medical Center Dinasty"
Sponsor class: OTHER
Collaborators: name: INBIO, LLC
First posted: 2019-09-20
Enrollment: 150
Sex: ALL
Minimum age (years): 1
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: In total, the study will involve 150 patients in three groups. The first and second groups will receive infusions of umbilical cord blood hematopoietic cells, with varying degrees of selection for the genes of the HLA system.

The third group will be the control group. In the control group, 50 patients with similar pathology will be examined, comparable in age, sex and degree of disorder of the type of "paracopies" against the background of standard therapy.
Primary purpose: TREATMENT

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.

RESEARCH PURPOSES

1. To develop an algorithm of using the method of transfusion of hematopoietic cells of umbilical cord blood at complex therapy of patients with children's cerebral palsy.
2. Formulate criteria for selecting patients for this method.
3. To analyze the safety and efficacy of umbilical cord blood hematopoietic cells transfusion in patients with cerebral palsy using evaluation scales.
4. To compare the efficacy of treatment of patients depending on the degree of tissue compatibility of the donor and recipient and other laboratory indications.
5. To implement the method of transfusion of umbilical cord blood hematopoietic cells in the complex therapy of patients with cerebral palsy.

Eligibility 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)

Patient 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.

Primary outcomes:
measure: Number of participants with non-serious and serious adverse events
timeFrame: 1 year
measure: Gross Motor Function Classification System (GMFCS - ER) scale severity change
timeFrame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
measure: Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.
timeFrame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
measure: Changes in The Infant Toddler Quality of Life Questionnaire for child above 3yrs.
timeFrame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
measure: Changes in Ashworth scale score for all child.
timeFrame: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)
Secondary outcomes:
measure: Number of Participants with Chimerism (longevity) of infused cell
timeFrame: Baseline, 6 month after first infusion
measure: The correlation between the concentration of CD14+ cells in the sample of UCB and the treatment effectiveness degree.
timeFrame: 1, 6 months (At each infusion)
measure: The correlation between the concentration of CD34+ cells in the UCB and the treatment effectiveness degree
timeFrame: 1, 6 months (At each infusion)
Other outcomes:
Locations:
city: Samara
country: Russian Federation
facility: Medical Centre Dinasty
Study officials:
affiliation: Medical Centre Dinasty
name: STANISLAV VOLCHKOV, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: