Estimation of the Safety and Efficiency Transfusion of HLA Matched CBU in Patients With CP

NCT ID: NCT04098029

Last Updated: 2023-01-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

150 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-09-01

Study Completion Date

2024-05-26

Brief Summary

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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

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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.

Conditions

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Cerebral Palsy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Group 1 Low HLA compatibility

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.

Group Type EXPERIMENTAL

Low HLA group CBU infusion

Intervention Type BIOLOGICAL

CB-MNC (Cord Blood Mononuclear Cells) infusion from different donors. One dose consists of 2-15х10\^7 cells per 1 kg of patient weight for each infusion. The protocol includes 2 infusions with an interval of 6-x months. HLA compatibility of CB is 3 or fewer by A, B, DRB1 loci.

Group 2 High HLA compatibility

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.

Group Type EXPERIMENTAL

High HLA group CBU infusion

Intervention Type BIOLOGICAL

CB-MNC (Cord Blood Mononuclear Cells) infusion from different donors. One dose consists of 2-15х10\^7 cells per 1 kg of patient weight for each infusion. The protocol includes 2 infusions with an interval of 6-x months. HLA compatibility of CB is 4 or more by A, B, DRB1 loci.

Standard therapy

Patients with standard therapy as a control group

Group Type OTHER

Standard therapy

Intervention Type OTHER

The standard therapy can include drugs, special psychology training, etc.

Interventions

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Low HLA group CBU infusion

CB-MNC (Cord Blood Mononuclear Cells) infusion from different donors. One dose consists of 2-15х10\^7 cells per 1 kg of patient weight for each infusion. The protocol includes 2 infusions with an interval of 6-x months. HLA compatibility of CB is 3 or fewer by A, B, DRB1 loci.

Intervention Type BIOLOGICAL

High HLA group CBU infusion

CB-MNC (Cord Blood Mononuclear Cells) infusion from different donors. One dose consists of 2-15х10\^7 cells per 1 kg of patient weight for each infusion. The protocol includes 2 infusions with an interval of 6-x months. HLA compatibility of CB is 4 or more by A, B, DRB1 loci.

Intervention Type BIOLOGICAL

Standard therapy

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Intervention Type OTHER

Other Intervention Names

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Control group

Eligibility Criteria

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Exclusion Criteria

* 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.
Minimum Eligible Age

1 Year

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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INBIO, LLC

UNKNOWN

Sponsor Role collaborator

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

OTHER

Sponsor Role lead

Responsible Party

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Volchkov Stanislav

Deputy director, Quality assurance director

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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STANISLAV VOLCHKOV, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Medical Centre Dinasty

Locations

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Medical Centre Dinasty

Samara, , Russia

Site Status RECRUITING

Countries

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Russia

Central Contacts

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STANISLAV VOLCHKOV, MD, PhD

Role: CONTACT

+79608159408

Olga Tyumina, M.D, PhD

Role: CONTACT

Facility Contacts

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Stanislav Volchkov, MD, PhD

Role: primary

+79277811532

Olga Tyumina, MD, PhD

Role: backup

+78469564455

Other Identifiers

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CP-HLA2019

Identifier Type: -

Identifier Source: org_study_id

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