Estimation of the Safety and Efficiency Transfusion of HLA Matched CBU in Patients With CP (CP-HLA2019)

State-Financed Health Facility "Samara Regional Medical Center Dinasty"

Status and phase: Enrolling; Phase 2

Conditions: Cerebral Palsy

Treatments: Biological: High HLA group CBU infusion; Other: Standard therapy; Biological: Low HLA group CBU infusion

Study type: Interventional

Funder types: Other

Identifiers

NCT04098029

CP-HLA2019

Details and patient eligibility

About

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

Full 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.

Enrollment: 150 estimated patients

Sex: All

Ages: 1 to 12 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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150 participants in 3 patient groups

Group 1 Low HLA compatibility

Experimental group

Description:

The patients in the first group will receive two CBU of low-level HLA matched infusions within a 6-month interval. The low-level match is 3 or less HLA compatibility degree by A, B, DRB1 loci.

Treatment:

Biological: Low HLA group CBU infusion

Group 2 High HLA compatibility

Experimental group

Description:

The patients in the second group will receive two CBU of high-level HLA matched infusions within a 6-month interval. The high-level match is 4 or more HLA compatibility degree by A, B, DRB1 loci.

Treatment:

Biological: High HLA group CBU infusion

Standard therapy

Other group

Description:

Patients with standard therapy as a control group

Treatment:

Other: Standard therapy