Autism Spectrum DisorderPhase 1/2ASD-HLA2019NCT04099381
Safety and Efficacy of the Transfusion of UCB in Patients With an ASD Depending on the Degree of HLA Compatibility.
A Phase 1/2 study testing ASD CB-MNC infusion low level HLA compatibility., ASD CB-MNC infusion high level HLA compatibility. and Standard therapy. in people with Autism Spectrum Disorder and Autism, run by State-Financed Health Facility "Samara Regional Medical Center Dinasty" at 1 site in Russia.
Trial summary
Autism is one of those disorders in Autism spectrum disorders (ASD), which characterized by social interaction abnormalities, impaired verbal and non-verbal communication, and repetitive, obsessive behavior, while the therapeutic effect of current treatments remains limited progress. Read more about this study
- Who is it for?
- Ages 3 to 14 years
- Conditions the study is for
- Autism Spectrum Disorder, Autism
- What is being tested?
- ASD CB-MNC infusion low level HLA compatibility., ASD CB-MNC infusion high level HLA compatibility. 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
- 10 Aug 2019to 26 May 2024
- Phase
- Phase 1/2
- 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 3 to 14 years
- All sexes
- No healthy volunteers
- Child
Patient selection criteria (indications for this type of treatment):
- Patient's age from 4 to 14 years;
- Diagnosis: autistic spectrum disorder;
- The severity of the disease on the ATEC scale of at least 16 points;
- The presence of a compatible allogeneic sample suitable for infusion;
- Parental consent (official guardians).
Patient exclusion criteria (contraindications for this type of treatment):
- The patient's age under 4 years, after 14 years;
- The presence of the following diseases in the history: heart failure at the stage of decompensation, stroke in the history of less than 1 year ago, anemia and other blood diseases;
- Decompensation for chronic and endocrinological diseases;
- Acute viral and bacterial infections during the acute clinical phase of the disease;
- HIV infection, hepatitis B and C;
- Cancer, chemotherapy, and history of cancer;
- Tuberculosis;
- Severe form of intellectual disability as a concomitant disease (diagnosis can be ignored, according to the decision of the Medical Committee of the Center);
- Fragile X chromosome syndrome;
- 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.
Olga Tyumina, M.D, PhD contact
Russia1 site, 1 recruiting
- Recruiting
Medical Centre Dinasty
Samara, 443095
Stanislav Volchkov, MD, PhD, +79277811532, ct@cordbank.ru
Olga Tyumina, MD, PhD, +78469564455
About this study
Official title: Protocol for Evaluating the Safety and Efficacy of Transfusion of Umbilical Cord Blood in Patients With a Diagnosis of Autism Spectrum Disorder Depending on the Degree of Tissue Compatibility of the Donor and the Recipient
Autism is one of those disorders in Autism spectrum disorders (ASD), which characterized by social interaction abnormalities, impaired verbal and non-verbal communication, and repetitive, obsessive behavior, while the therapeutic effect of current treatments remains limited progress.
The possible reason for ASD is neural hypoperfusion and immune dysregulation. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. In this study, the safety and efficacy of hUCB-MNCs infusion will be evaluated in patients with Autism with regarding to HLA compatibility.
Read the detailed description
Autism Spectrum Disorders (ASD) are heterogeneous neuro-developed disorders. Autism is the most common disorder of the diseases characterized by dysfunctions in response to social interaction and communication, as well as the presence of repetitive and stereotyped behaviors. Recent reports of a sharp increase in the number of children with autism. The exact etiology of autism remains unclear. Compliance, the definition of effective treatments for autism is particularly difficult.
Although it has been understood, it can be a question of immune dysregulation. Examination of the inflammatory cytokines, dysfunction of the immune system and the immune system. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. Our study suggest that infusion of cord blood mononuclear cells will affect Autism.
This protocol was developed on the basis of the results of the previously approved protocol of the center NCT03786744 (Transfusion of allogeneic cord blood samples in patients with autism spectrum disorders), 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.
- Conditions
- Autism Spectrum Disorder, Autism
- Being tested
- ASD CB-MNC infusion low level HLA compatibility., ASD CB-MNC infusion high level HLA compatibility., Standard therapy.
- Keywords
- Autism, ASD, Cord blood, HLA
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASD CB-MNC infusion low level HLA compatibility.Biological
CB-MNC 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 the cord blood is 3 or fewer by A, B, DRB1 loci.
ASD CB-MNC infusion high level HLA compatibility.Biological
CB-MNC 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 the cord blood is 4 or more by A, B, DRB1 loci.
Standard therapy.Other
The standard therapy can include drugs, special psychology training etc.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 Low HLA compatibility | Experimental | ASD CB-MNC infusion from different donors and standard therapy. CBU with 3 or less HLA compatibility degree in A, B, DRB1 loci will be used. Receives: Biological: ASD CB-MNC infusion low level HLA compatibility. |
| Group 2 High HLA compatibility | Experimental | ASD CB-MNC infusion from different donors and standard therapy. CBU with 3 or more HLA compatibility degree in A, B, DRB1 loci will be used. Receives: Biological: ASD CB-MNC infusion high level HLA compatibility. |
| Group 3 Control | Other | Patients 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.
Number of participants with non-serious and serious adverse events.
Safety assessment. Adverse events rate will be assessed in all patients.
Measured: baseline, change from baseline at 12 month
The Childhood Autism Rating Scale (CARS2).
Rating for behavior, characteristics, and abilities against the expected developmental growth. Will be assessed fifteen items, including: relationship to people; imitation; emotional response; body; object use; adaptation to change; visual response; listening response; taste-smell-touch response and use; fear and nervousness; verbal communication; non-verbal communication; activity level; level and consistency of intellectual response; general impressions. Total score will be assessed. Scores range form 15 to 60 with 30 being the cutoff rate for a diagnosis of mild autism. Scores 30-37 indicate mild to moderate autism, while scores between 38 and 60 are characterized as severe autism.
Measured: baseline, change from baseline at 6,12 month
Autism Treatment Evaluation Checklist (ATEC).
Will be assessed: I. Speech/Language Communication (14 items); II. Sociability (20 items); III. Sensory/ Cognitive Awareness (18 items); and IV. Health/Physical/Behavior (25 items).
Measured: baseline, change from baseline every month (total 12 evaluations)
Secondary outcomes (4)
Evaluation of the survival of donor cells in the host body without immune responses
Evaluation of the survival of donor cells in the host body to determine the safety and duration of the cellular component. To do this, before the first injection of the cells, a sample of the patient's blood and a donor cord blood sample will be taken. 6 months after the first injection of the cells, immediately before the second injection, the patient's blood will be re-taken to conduct a study on chimerism. The obtained data will allow assessing the survival degree of donor material. The definition of chimerism is carried out by fragment analysis. For analysis, blood samples of the biomaterial will be collected from the patient before the cells are injected and 6 months after the injection.
Measured: baseline, change from baseline at 6 month
The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the sample of umbilical cord blood and the treatment effectiveness degree
A correlation between the concentration of cells of the monocyte-macrophage series (CD4/CD25/FoxP3) and the degree of recovery of neurological functions. 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: At 1, at 6 months (At first and second infusion)
The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the patient depending on the severity of the disease.
A correlation between the concentration of T regulatory cells (CD4/CD25/FoxP3) and the severity of the disease. 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 first injection of the cells, and before the second injection of the cells.
Measured: At 1, at 6 months (At first and second infusion)
The correlation of the concentration of CD34+ cells in the cord blood sample and the treatment effectiveness degree
CD34+ cells are precursors of hematopoiesis and endothelium. Studies show that CD34+ cells, when injected into an ischemic lesion zone, exert protective properties on the affected cells, stimulate angiogenesis, and are involved in reducing inflammatory reactions. In this study, we plan to find a correlation between the concentration of the introduction of CD34+ cells and 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: At 1, at 6 months (At first and second infusion)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2019
- Study start10 Aug 2019
- Primary completion26 Dec 2023 (estimated)
- Study completion26 May 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- This is prospective, non-randomized (open-label) with control group study. In total, the study will involve 150 patients in three groups. The first and second groups will receive transfusions 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 "para-copies" against the background of standard therapy.
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- State-Financed Health Facility "Samara Regional Medical Center Dinasty"
- Sponsor type
- University, hospital or other
- Collaborators
- INBIO, LLC
- Responsible party
- Principal investigator, Volchkov Stanislav, State-Financed Health Facility "Samara Regional Medical Center Dinasty"
- Investigators
- STANISLAV VOLCHKOV, MD, PhD, Principal investigator, Medical Centre Dinasty
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04099381
- Sponsor's protocol ID
- ASD-HLA2019
- Acronym
- ASD-HLA2019
- Status last verified
- Jan 2023
- First submitted
- 17 Sep 2019
- Last updated
- 18 Jan 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protocol for Evaluating the Safety and Efficacy of Transfusion of Umbilical Cord Blood in Patients With a Diagnosis of Autism Spectrum Disorder Depending on the Degree of Tissue Compatibility of the Donor and the Recipient
- ClinicalTrials.gov ID
- NCT04099381
- Acronym / sponsor ID
- ASD-HLA2019 / ASD-HLA2019
- Conditions
- Autism Spectrum Disorder, Autism
- Keywords
- Autism, ASD, Cord blood, HLA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- State-Financed Health Facility "Samara Regional Medical Center Dinasty"University, hospital or other
- Collaborators
- INBIO, LLC
- Study start
- 10 Aug 2019 (actual)
- Primary completion
- 26 Dec 2023 (estimated)
- Study completion
- 26 May 2024 (estimated)
- First posted
- 23 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Autism is one of those disorders in Autism spectrum disorders (ASD), which characterized by social interaction abnormalities, impaired verbal and non-verbal communication, and repetitive, obsessive behavior, while the therapeutic effect of current treatments remains limited progress.
The possible reason for ASD is neural hypoperfusion and immune dysregulation. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. In this study, the safety and efficacy of hUCB-MNCs infusion will be evaluated in patients with Autism with regarding to HLA compatibility.
Detailed description
Autism Spectrum Disorders (ASD) are heterogeneous neuro-developed disorders. Autism is the most common disorder of the diseases characterized by dysfunctions in response to social interaction and communication, as well as the presence of repetitive and stereotyped behaviors. Recent reports of a sharp increase in the number of children with autism. The exact etiology of autism remains unclear. Compliance, the definition of effective treatments for autism is particularly difficult.
Although it has been understood, it can be a question of immune dysregulation. Examination of the inflammatory cytokines, dysfunction of the immune system and the immune system. The Human Umbilical Cord Blood Mononuclear Cells (hUCB-MNCs) have been shown to have the ability to modulate the immune response and enhance angiogenesis, suggesting the novel and promising therapeutic strategy. Our study suggest that infusion of cord blood mononuclear cells will affect Autism.
This protocol was developed on the basis of the results of the previously approved protocol of the center NCT03786744 (Transfusion of allogeneic cord blood samples in patients with autism spectrum disorders), 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- This is prospective, non-randomized (open-label) with control group study. In total, the study will involve 150 patients in three groups. The first and second groups will receive transfusions 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 "para-copies" against the background of standard therapy.
| Arm | Type | Receives |
|---|---|---|
| Group 1 Low HLA compatibility | Experimental | Biological: ASD CB-MNC infusion low level HLA compatibility.ASD CB-MNC infusion from different donors and standard therapy. CBU with 3 or less HLA compatibility degree in A, B, DRB1 loci will be used. |
| Group 2 High HLA compatibility | Experimental | Biological: ASD CB-MNC infusion high level HLA compatibility.ASD CB-MNC infusion from different donors and standard therapy. CBU with 3 or more HLA compatibility degree in A, B, DRB1 loci will be used. |
| Group 3 Control | Other | Other: Standard therapy.Patients with standard therapy as a control group. |
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ASD CB-MNC infusion low level HLA compatibility.CB-MNC 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 the cord blood is 3 or fewer by A, B, DRB1 loci. | Biological | Group 1 Low HLA compatibility |
| ASD CB-MNC infusion high level HLA compatibility.CB-MNC 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 the cord blood is 4 or more by A, B, DRB1 loci. | Biological | Group 2 High HLA compatibility |
| Standard therapy.The standard therapy can include drugs, special psychology training etc. | Other | Group 3 Control |
Primary outcome
Number of participants with non-serious and serious adverse events.
Time frame: baseline, change from baseline at 12 month
Safety assessment. Adverse events rate will be assessed in all patients.
The Childhood Autism Rating Scale (CARS2).
Time frame: baseline, change from baseline at 6,12 month
Rating for behavior, characteristics, and abilities against the expected developmental growth. Will be assessed fifteen items, including: relationship to people; imitation; emotional response; body; object use; adaptation to change; visual response; listening response; taste-smell-touch response and use; fear and nervousness; verbal communication; non-verbal communication; activity level; level and consistency of intellectual response; general impressions. Total score will be assessed. Scores range form 15 to 60 with 30 being the cutoff rate for a diagnosis of mild autism. Scores 30-37 indicate mild to moderate autism, while scores between 38 and 60 are characterized as severe autism.
Autism Treatment Evaluation Checklist (ATEC).
Time frame: baseline, change from baseline every month (total 12 evaluations)
Will be assessed: I. Speech/Language Communication (14 items); II. Sociability (20 items); III. Sensory/ Cognitive Awareness (18 items); and IV. Health/Physical/Behavior (25 items).
4 secondary outcomes
Evaluation of the survival of donor cells in the host body without immune responses
Time frame: baseline, change from baseline at 6 month
The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the sample of umbilical cord blood and the treatment effectiveness degree
Time frame: At 1, at 6 months (At first and second infusion)
The correlation of the concentration of T-regulatory cells (CD4/CD25/FoxP3) in the patient depending on the severity of the disease.
Time frame: At 1, at 6 months (At first and second infusion)
The correlation of the concentration of CD34+ cells in the cord blood sample and the treatment effectiveness degree
Time frame: At 1, at 6 months (At first and second infusion)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Olga Tyumina, M.D, PhD contact
STANISLAV VOLCHKOV, MD, PhD principal investigator
Medical Centre Dinasty
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ASD CB-MNC infusion low level HLA compatibility., ASD CB-MNC infusion high level HLA compatibility. and Standard therapy. in people with Autism Spectrum Disorder and Autism, 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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, bioen07@gmail.com), 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: baseline, change from baseline at 12 month). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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.