Autism Spectrum DisorderPhase 1/2NCT03786744
Allogenic Cord Blood Transfusion in Patients With Autism
A Phase 1/2 study testing ASD CB-MNC injection. 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 15 years
- Conditions the study is for
- Autism Spectrum Disorder, Autism
- What is being tested?
- ASD CB-MNC injection. and Standard therapy.
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteRussia
- Study dates
- 12 Oct 2018to 12 Dec 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Confirmed diagnosis: autistic type disorder (ASD).
- Systemic speech underdevelopment
- The presence of attention deficit hyperactivity disorder as a comorbid state
- Cognitive impairment
Who cannot join
- organic pathology of the brain according to CT, MRI
- the presence of the following diseases in history: heart failure in the stage of decompensation, stroke in history less than 1 year ago, blood diseases;
- decompensation of chronic and endocrinological diseases;
- acute respiratory viral and bacterial infections, period less than 1 month after the acute phase.
- HIV infection, hepatitis B and C.
- oncological diseases, chemotherapy in the anamnesis;
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- tuberculosis.
- Severe form of intellectual disability.
- Cerebral palsy.
- epilepsy.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficiency Evaluation of Allogenic Umbilical Cord Blood (UCB) Transfusion in Patients With Autism
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.
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 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.
- Conditions
- Autism Spectrum Disorder, Autism
- Being tested
- ASD CB-MNC injection., Standard therapy.
- Keywords
- Autism, ASD, Cord blood
- 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 injection.Biological
CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals.
Standard therapy.Other
The standard therapy can include drugs, special psychology training etc.
| Study group | Role | What they receive |
|---|---|---|
| ASD CB-MNC injection. | Experimental | ASD CB-MNC injection from different donors and standard therapy. Receives: Biological: ASD CB-MNC injection.; Other: Standard therapy. |
| Standard therapy. | Other | Patients with standard therapy as control group. Receives: Other: Standard therapy. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: score at baseline, 1, 2, 6, 12 months
Secondary outcomes (3)
Cytokine Analysis.
IL1b, IL6, TNF-alpha, IL8, y-IFN measure in patient peripheral blood
Measured: score at baseline, 1, 2, 6 months
Immune Status as Determined by Percentage of Peripheral Blood Cells
Peripheral blood flow cytometry cell count for CD3; CD4; CD8; CD19; CD16-56 (%)
Measured: 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.)
Measured: score at baseline, 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Dec 2018
- Study start12 Oct 2018
- Primary completion12 Oct 2019
- Study completion12 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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. 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).
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- State-Financed Health Facility "Samara Regional Medical Center Dinasty"
- Sponsor type
- University, hospital or other
- 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
- NCT03786744
- Sponsor's protocol ID
- 12112018
- Status last verified
- Oct 2020
- First submitted
- 21 Dec 2018
- Last updated
- 19 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficiency Evaluation of Allogenic Umbilical Cord Blood (UCB) Transfusion in Patients With Autism
- ClinicalTrials.gov ID
- NCT03786744
- Acronym / sponsor ID
- 12112018
- Conditions
- Autism Spectrum Disorder, Autism
- Keywords
- Autism, ASD, Cord blood
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- State-Financed Health Facility "Samara Regional Medical Center Dinasty"University, hospital or other
- Study start
- 12 Oct 2018 (actual)
- Primary completion
- 12 Oct 2019 (actual)
- Study completion
- 12 Dec 2019 (actual)
- First posted
- 26 Dec 2018 (actual)
- Results
- Posted 19 Nov 2020
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 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.
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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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).
| Arm | Type | Receives |
|---|---|---|
| ASD CB-MNC injection. | Experimental | Biological: ASD CB-MNC injection., Other: Standard therapy.ASD CB-MNC injection from different donors and standard therapy. |
| Standard therapy. | Other | Other: Standard therapy.Patients with standard therapy as control group. |
Who may qualify
- Ages
- 3 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Confirmed diagnosis: autistic type disorder (ASD).
- Systemic speech underdevelopment
- The presence of attention deficit hyperactivity disorder as a comorbid state
- Cognitive impairment
Key exclusion criteria (10 listed)
- organic pathology of the brain according to CT, MRI
- the presence of the following diseases in history: heart failure in the stage of decompensation, stroke in history less than 1 year ago, blood diseases;
- decompensation of chronic and endocrinological diseases;
- acute respiratory viral and bacterial infections, period less than 1 month after the acute phase.
- HIV infection, hepatitis B and C.
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 injection.CB-MNC injection from different donors. One dose consist 20-50 mil MNC/kg.The protocol include 3 injection at monthly intervals. | Biological | ASD CB-MNC injection. |
| Standard therapy.The standard therapy can include drugs, special psychology training etc. | Other | ASD CB-MNC injection., Standard therapy. |
Primary outcome
A Number of Participants With Non-serious and Serious Adverse Events 24 Hrs Post-transfusion
Time frame: 24hrs post transfusion
Safety assessment. The adverse events rate will be assessed in all patients.
Autism Treatment Evaluation Checklist (ATEC).
Time frame: score at baseline, 1, 2, 6, 12 months
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.
3 secondary outcomes
Cytokine Analysis.
Time frame: score at baseline, 1, 2, 6 months
Immune Status as Determined by Percentage of Peripheral Blood Cells
Time frame: score at baseline, 6 month
Immune Status as Determined by Absolute Cell Count of Peripheral Blood Cells
Time frame: score at baseline, 6 month
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
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 injection. 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?
The sponsor lists 4 inclusion and 10 exclusion criteria. 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: finished, as of the sponsor's last update on 19 Nov 2020. 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?
Yes. Results are posted in the registry (first posted 19 Nov 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
A Number of Participants With Non-serious and Serious Adverse Events 24 Hrs Post-transfusion (time frame: 24hrs post transfusion). The study lists 2 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 19 Nov 2020.