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Cerebral PalsyPhase 2CP-HLA2019NCT04098029

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

A Phase 2 study testing Low HLA group CBU infusion, High HLA group CBU infusion and Standard therapy in people with Cerebral Palsy, run by State-Financed Health Facility "Samara Regional Medical Center Dinasty" at 1 site in Russia.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 12 years
Conditions the study is for
Cerebral Palsy
What is being tested?
Low HLA group CBU infusion, High HLA group CBU infusion and Standard therapy
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
150 participants
Where
1 siteRussia
Study dates
1 Sep 2019to 26 May 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 12 years
  • All sexes
  • No healthy volunteers
  • Child

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 locations and contacts

1 site in 1 country.

STANISLAV VOLCHKOV, MD, PhD contact

+79608159408

ct@cordbank.ru

Olga Tyumina, M.D, PhD contact

centr123@bk.ru

Russia1 site, 1 recruiting

  • Medical Centre Dinasty

    Samara, 443095

    Stanislav Volchkov, MD, PhD, +79277811532, ct@cordbank.ru

    Olga Tyumina, MD, PhD, +78469564455

    Recruiting

Other trials in Russia

About this study

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

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.

Read more

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

Read the 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.
Conditions
Cerebral Palsy
Being tested
Low HLA group CBU infusion, High HLA group CBU infusion, Standard therapy
Keywords
Cerebral palsy, CP, Cord Blood, Stem cells, HLA
Medical subject headings
Cerebral Palsy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Low HLA group CBU infusionBiological

    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.

  • High HLA group CBU infusionBiological

    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 therapyOther

    Also called Control group

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

Study groups in NCT04098029
Study groupRoleWhat they receive
Group 1 Low HLA compatibilityExperimentalThe 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.
Receives: Biological: Low HLA group CBU infusion
Group 2 High HLA compatibilityExperimentalThe 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.
Receives: Biological: High HLA group CBU infusion
Standard therapyOtherPatients with standard therapy as a control group
Receives: Other: Standard therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with non-serious and serious adverse events

    Safety assessment such as adverse events will be registered. Adverse events will be monitored during all trial.

    Measured: 1 year

  2. Gross Motor Function Classification System (GMFCS - ER) scale severity change

    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.

    Measured: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)

  3. Changes in Standardized Gross Motor Function 66 (GMFM-66) Score for all child.

    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.

    Measured: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)

  4. Changes in The Infant Toddler Quality of Life Questionnaire for child above 3yrs.

    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.

    Measured: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)

  5. Changes in Ashworth scale score for all child.

    The Ashworth scale (AS) measures resistance during passive soft-tissue stretching and is used as a simple measure of spasticity scoring, where:

    1. No increase in tone;
    2. A slight increase in tone giving catch when the limb is moved in flexion and extension;
    3. A more marked increase in tone, but the limb is easily flexed;
    4. Considerable increases in tone, passive movement difficult;
    5. Limb rigid in flexion or extension. This test will be acquired for the child above 3yrs.

    Measured: Baseline, 6 month after first infusion, 6 month after second infusion (3 times)

Secondary outcomes (3)

  1. Number of Participants with Chimerism (longevity) of infused cell

    Chimerism study to detect the longevity of infused donor cells and predict the effectiveness of treatment. This study measures the DNA of donor cells will in patients' blood at the 6-month post-infusion. The absence of donor DNA suggests no chimerism.

    Measured: Baseline, 6 month after first infusion

  2. The correlation between the concentration of CD14+ cells in the sample of UCB and the treatment effectiveness degree.

    The correlation between the concentration of cells of the monocyte-macrophage series (CD14+) and the degree of recovery of neurological functions. Data from assessments scales with progression rate will be compared with concentration of CD14+ in infused CBU. This information will be used to assess treatment effectiveness. The assessment of a cord blood sample will be carried out on a flow cytometer immediately before the injection of the cells or in the next 12 hours.

    Measured: 1, 6 months (At each infusion)

  3. The correlation between the concentration of CD34+ cells in the UCB and the treatment effectiveness degree

    A correlation between the concentration of CD34+ cells and the degree of recovery of neurological functions. Data from assessments scales with progression rate will be compared with concentration of CD34+ cells of infused CBU. The assessment of a cord blood sample will be carried out on a flow cytometer immediately before the injection of the cells or in the next 12 hours.

    Measured: 1, 6 months (At each infusion)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Sep 2019
  2. Study start1 Sep 2019
  3. Primary completion26 Dec 2023 (estimated)
  4. Study completion26 May 2024 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
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.
Masking
None (open label)
Enrolment
150 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04098029
Sponsor's protocol ID
CP-HLA2019
Acronym
CP-HLA2019
Status last verified
Jan 2023
First submitted
17 Sep 2019
Last updated
18 Jan 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Low HLA group CBU infusion, High HLA group CBU infusion and Standard therapy in people with Cerebral Palsy, run by State-Financed Health Facility "Samara Regional Medical Center Dinasty" at 1 site in Russia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 18 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact STANISLAV VOLCHKOV, MD, PhD (+79608159408, ct@cordbank.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with non-serious and serious adverse events (time frame: 1 year). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04098029.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jan 2023.

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