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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 deregulation. 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.
Autism Spectrum Disorders (ASD) are heterogeneous neuro-developed disorders. Autism is the most common disorder of the diseases characterized by dysfunctions in response 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. The Our study suggest that infusion of cord blood mononuclear cells will affect Autism.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ASD CB-MNC injection. | Experimental | ASD CB-MNC injection from different donors and standard therapy. |
|
| Standard therapy. | Other | Patients with standard therapy as control group. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ASD CB-MNC injection. | Biological | CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals. |
|
| Measure | Description | Time Frame |
|---|---|---|
| A Number of Participants With Non-serious and Serious Adverse Events 24 Hrs Post-transfusion | Safety assessment. The adverse events rate will be assessed in all patients. | 24hrs post transfusion |
| Autism Treatment Evaluation Checklist (ATEC). | The ATEC is designed to measure changes in the severity of ASD in response to treatment. A total score and 4 subscale scores are reported. Questions in the first 3 subscales are scored using a 0-3 scale (not true/partially true/absolutely true). The 4 subscale, Health/Physical/Behavior, is scored using a 0-4 point scale (Not a problem/Minor problem/Moderate problem/Serious problem). The first subscale, Speech/Language/Communication, contains 14 items where the score ranges from 0-28 points. The Sociability subscale contains 20 items and participants can score from 0-40. The third subscale, Sensory/Cognitive awareness, has 18 items and scores range from 0-36. Finally, the Health/Physical/Behavior subscale contains 25 items and scores range from 0-75. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD. | score at baseline, 1, 2, 6, 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Cytokine Analysis. | IL1b, IL6, TNF-alpha, IL8, y-IFN measure in patient peripheral blood | score at baseline, 1, 2, 6 months |
| Immune Status as Determined by Percentage of Peripheral Blood Cells |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| STANISLAV VOLCHKOV, MD, PhD | Medical Centre Dinasty | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical Centre Dinasty | Samara | 443095 | Russia |
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| ID | Title | Description |
|---|---|---|
| FG000 | ASD CB-MNC Injection. | ASD CB-MNC injection from different donors and standard therapy. ASD CB-MNC injection.: CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals. Standard therapy.: The standard therapy can include drugs, special psychology training etc. |
| FG001 | Standard Therapy. | Patients with standard therapy as control group. Standard therapy.: The standard therapy can include drugs, special psychology training etc. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ASD CB-MNC Injection. | ASD CB-MNC injection from different donors and standard therapy. ASD CB-MNC injection.: CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals. Standard therapy.: The standard therapy can include drugs, special psychology training etc. |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | A Number of Participants With Non-serious and Serious Adverse Events 24 Hrs Post-transfusion | Safety assessment. The adverse events rate will be assessed in all patients. | Posted | Number | participants | 24hrs post transfusion |
|
12 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ASD CB-MNC Injection. | ASD CB-MNC injection from different donors and standard therapy. ASD CB-MNC injection.: CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals. Standard therapy.: The standard therapy can include drugs, special psychology training etc. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infectious nature | Cardiac disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Volchkov Stanislav | Samara Medical Center Dinasty | +78469564455 | quality@cordbank.ru |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 12, 2018 | Aug 14, 2020 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| D001321 | Autistic Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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This is prospective, non-randomized (open label) with control group study. The total 20 patient will be enrolled in this study. In 10 patient with ASD will be infused with umbilical corb blood. Other 10 patient with ASD will be treated with standard therapy (control group).
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| Standard therapy. | Other | The standard therapy can include drugs, special psychology training etc. |
|
Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (%)
| score at baseline, 6 month |
| Immune Status as Determined by Absolute Cell Count of Peripheral Blood Cells | Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (absolute v.) | score at baseline, 6 month |
| Standard Therapy. |
Patients with standard therapy as control group. Standard therapy.: The standard therapy can include drugs, special psychology training etc. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Primary | Autism Treatment Evaluation Checklist (ATEC). | The ATEC is designed to measure changes in the severity of ASD in response to treatment. A total score and 4 subscale scores are reported. Questions in the first 3 subscales are scored using a 0-3 scale (not true/partially true/absolutely true). The 4 subscale, Health/Physical/Behavior, is scored using a 0-4 point scale (Not a problem/Minor problem/Moderate problem/Serious problem). The first subscale, Speech/Language/Communication, contains 14 items where the score ranges from 0-28 points. The Sociability subscale contains 20 items and participants can score from 0-40. The third subscale, Sensory/Cognitive awareness, has 18 items and scores range from 0-36. Finally, the Health/Physical/Behavior subscale contains 25 items and scores range from 0-75. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD. | Posted | Median | Inter-Quartile Range | score on a scale | score at baseline, 1, 2, 6, 12 months |
|
|
|
|
| Secondary | Cytokine Analysis. | IL1b, IL6, TNF-alpha, IL8, y-IFN measure in patient peripheral blood | Posted | Median | Inter-Quartile Range | pg/mL | score at baseline, 1, 2, 6 months |
|
|
|
| Secondary | Immune Status as Determined by Percentage of Peripheral Blood Cells | Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (%) | Posted | Median | Inter-Quartile Range | percentage of cells | score at baseline, 6 month |
|
|
|
| Secondary | Immune Status as Determined by Absolute Cell Count of Peripheral Blood Cells | Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (absolute v.) | Posted | Median | Inter-Quartile Range | cells/µl | score at baseline, 6 month |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
| 6 |
| 10 |
| EG001 | Standard Therapy. | Patients with standard therapy as control group. Standard therapy.: The standard therapy can include drugs, special psychology training etc. | 0 | 10 | 0 | 10 | 8 | 10 |
| Non Infectious nature | Cardiac disorders | Non-systematic Assessment |
|
| Infectious nature | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment | 3 cases of influenza
|
|
| NON Infectious nature | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Infectious nature | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| NON Infectious nature | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Infectious nature | Renal and urinary disorders | Non-systematic Assessment |
|
| NON Infectious nature | Renal and urinary disorders | Non-systematic Assessment |
|
| Infectious nature | Gastrointestinal disorders | Non-systematic Assessment | food toxicology |
|
| NON Infectious nature | Gastrointestinal disorders | Non-systematic Assessment | Gastritis |
|
| Infectious nature | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| NON Infectious nature | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Infectious nature | Nervous system disorders | Non-systematic Assessment |
|
| NON Infectious nature | Nervous system disorders | Non-systematic Assessment | head pain, dizziness |
|
| Psychiatric symptoms | Psychiatric disorders | Non-systematic Assessment | aggression irritability anxiety |
|
| Hypersensitivity | Immune system disorders | Non-systematic Assessment | seasonal allergy |
|
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| Speech/Language/Communication at 2 months |
|
| Speech/Language/Communication at 6 months |
|
| Speech/Language/Communication at 12 months |
|
| Sociability at baseline |
|
| Sociability at 1 months |
|
| Sociability at 2 months |
|
| Sociability at 6 months |
|
| Sociability at 12 months |
|
| Sensory/Cognitive Awareness at baseline |
|
| Sensory/Cognitive Awareness at 1 months |
|
| Sensory/Cognitive Awareness at 2 months |
|
| Sensory/Cognitive Awareness at 6 months |
|
| Sensory/Cognitive Awareness at 12 months |
|
| Health/ Physical Development/Behaviour at baseline |
|
| Health/ Physical Development/Behaviour at 1 months |
|
| Health/ Physical Development/Behaviour at 2 months |
|
| Health/ Physical Development/Behaviour at 6 months |
|
| Health/ Physical Development/Behaviour at 12months |
|
| Total score at baseline |
|
| Total score at 1 months |
|
| Total score at 2 months |
|
| Total score at 6 months |
|
| Total score at 12 months |
|
| Superiority |
| The study results were statistically processed using parametric and non-parametric methods using STATA version 9.0, Statistica for Windows version 6.0 and MS Office Excel 2007. Changes from the beginning of therapy and after 1, 2, 6, 12 months were compared using the Wilcoxon criterion, data between the main group and the control group were compared using non-parametric method, Mann-Whitney test was used (data are presented in Me - median format with an indication of 25% and 75% quartile. | Wilcoxon (Mann-Whitney) | 0.004072 | Alternative hypothesis: can be changed average of 2-month of Health/Physical Development/Behaviour for Cord Blood versus Control groups. Threshold <0.05 | Median Difference (Net) | 9 | 2-Sided | Superiority |
| The study results were statistically processed using parametric and non-parametric methods using STATA version 9.0, Statistica for Windows version 6.0 and MS Office Excel 2007. Changes from the beginning of therapy and after 1, 2, 6, 12 months were compared using the Wilcoxon criterion, data between the main group and the control group were compared using non-parametric method, Mann-Whitney test was used (data are presented in Me - median format with an indication of 25% and 75% quartile. | Wilcoxon (Mann-Whitney) | 0.017258 | Alternative hypothesis: can be changed average of 12-month of Health/Physical Development/Behaviour for Cord Blood versus Placebo groups. Threshold <0.05 | Mean Difference (Net) | 9 | 2-Sided | Superiority |
| The study results were statistically processed using parametric and non-parametric methods using STATA version 9.0, Statistica for Windows version 6.0. Changes from the beginning of therapy and after 1, 2, 6, 12 months were compared using the Wilcoxon criterion, data between the main group and the control group were compared using non-parametric method, Mann-Whitney test was used (data are presented in Me - median format with an indication of 25% and 75% quartile | Wilcoxon (Mann-Whitney) | 0.031210 | Alternative hypothesis: can be changed average of 1-month of total score for Cord Blood versus Placebo groups. Threshold <0.05 | Mean Difference (Net) | 10 | 2-Sided | Superiority |
| The study results were statistically processed using parametric and non-parametric methods using STATA version 9.0, Statistica for Windows version 6.0. Changes from the beginning of therapy and after 1, 2, 6, 12 months were compared using the Wilcoxon criterion, data between the main group and the control group were compared using non-parametric method, Mann-Whitney test was used (data are presented in Me - median format with an indication of 25% and 75% quartile | Wilcoxon (Mann-Whitney) | 0.001940 | Alternative hypothesis: can be changed average of 2-month of Total score for Cord Blood versus Placebo groups. Threshold <0.05 | Mean Difference (Net) | 24 | 2-Sided | Superiority |
| The study results were statistically processed using parametric and non-parametric methods using STATA version 9.0, Statistica for Windows version 6.0. Changes from the beginning of therapy and after 1, 2, 6, 12 months were compared using the Wilcoxon criterion, data between the main group and the control group were compared using non-parametric method, Mann-Whitney test was used (data are presented in Me - median format with an indication of 25% and 75% quartile | Wilcoxon (Mann-Whitney) | 0.031210 | Alternative hypothesis: can be changed average of 6-month of Total score for Cord Blood versus Placebo groups. Threshold <0.05 | Mean Difference (Net) | 15 | 2-Sided | Superiority |
| The study results were statistically processed using parametric and non-parametric methods using STATA version 9.0, Statistica for Windows version 6.0. Changes from the beginning of therapy and after 1, 2, 6, 12 months were compared using the Wilcoxon criterion, data between the main group and the control group were compared using non-parametric method, Mann-Whitney test was used (data are presented in Me - median format with an indication of 25% and 75% quartile | Wilcoxon (Mann-Whitney) | 0.011330 | Alternative hypothesis: can be changed average of 12-month of Total score for Cord Blood versus Placebo groups. Threshold <0.05 | Mean Difference (Net) | 16 | 2-Sided | Superiority |
| Title | Measurements |
|---|---|
|
| y-IFN pg/ml at 6 months |
|
| IL1b pg/ml at baseline |
|
| IL1b pg/ml at 1 months |
|
| IL1b pg/ml at 2 months |
|
| IL1b pg/ml at 6 months |
|
| IL6 pg/ml at baseline |
|
| IL6 pg/ml at 1 months |
|
| IL6 pg/ml at 2 months |
|
| IL6pg/ml at 6 months |
|
| IL8 pg/ml at baseline |
|
| IL8 pg/ml at 1 months |
|
| IL8 pg/ml at 2 months |
|
| IL8 pg/ml at 6 months |
|
| TNF-alpha pg/ml at baseline |
|
| TNF-alpha pg/ml at 1 months |
|
| TNF-alpha pg/ml at 2 months |
|
| TNF-alpha pg/ml at 6 months |
|
|
| СD3+СD8+(%) at 6 months |
|
| СD3+СD4+ (%) at baseline |
|
| СD3+СD4+(%) at 6 months |
|
| СD3+СD4+СD8+ (%) at baseline |
|
| СD3+СD4+СD8+(%) at 6 months |
|
| СD16+СD56+(%) at baseline |
|
| СD16+СD56+(%) at 6 months |
|
| СD19+(%) at baseline |
|
| СD19+ (%) at 6 months |
|
|
| CD 3+CD8+ at 6 months |
|
| CD 3+CD4+ at baseline |
|
| CD 3+CD4+ at 6 months |
|
| CD 3+ CD4+CD8+ at baseline |
|
| CD 3+ CD4+CD8+ at 6 months |
|
| CD 16+CD56+ at baseline |
|
| CD 16+CD56+ at 6 months |
|
| СD19+ at baseline |
|
| СD19+ at 6 months |
|