Autism Spectrum DisorderPhase 1NCT04710810
UCB Stem Cells for Autism Spectrum Disorders
A Phase 1 study testing umbilical cord blood mononuclear stem cells in people with Autism Spectrum Disorder, run by St. Petersburg Bekhterev Research Psychoneurological Institute at 1 site in Russia.
Trial summary
Purpose. The goals of the study are to assess the safety of the intravenous infusion of umbilical cord blood (UCB) cells in patients with autism and to confirm changes in social/ communicative skills and cognitive functioning after four infusions of ABO/Rh-matched UCB stem cells. Material and methods. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- umbilical cord blood mononuclear stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteRussia
- Study dates
- 4 Nov 2019to 31 Jul 2021
- Phase
- Phase 1
- 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 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- diagnosed ASD by ICD-10 criteria
Who cannot join
- acute somatic diseases
- indication for surgical (neurosurgical) therapy
- acute or chronic infectious diseases
- history of acute allergic reactions
Trial locations and contacts
1 site in 1 country.
Russia1 site
St.-Petersburg Bekhterev Reserach Psychoneurological Institute
Saint Petersburg, Sankt-Peterburg, 192019
About this study
Official title: Transplantation of Human Allogenic Cord Blood Mononuclear Stem Cells in Autism: Safety and Efficacy of the Method
Purpose. The goals of the study are to assess the safety of the intravenous infusion of umbilical cord blood (UCB) cells in patients with autism and to confirm changes in social/ communicative skills and cognitive functioning after four infusions of ABO/Rh-matched UCB stem cells.
Material and methods. The sample comprises 30 patients (27 males, 3 females) aged between 3 and 11 years with ASD under the care of the National Medical Research Center for Psychiatry and Neurology (Saint-Petersburg, Russia). Participants are randomly assigned to either the control group (14 males, 1 female) or the experimental group (13 males, 2 females). The experimental group receives intravenous injections of UCB cells four times with a two-week gap between injections. The control group receives standard therapy.
The dynamic of cognitive functions and social/communicative skills assess with Checklist for autism spectrum disorders (CASD), Autism treatment evaluation checklist (ATEC), subscales of Wechsler Intelligence Scale for Children (WISC) - "Digit Span", "Picture completion", "Block design", "Coding".
- Conditions
- Autism Spectrum Disorder
- Being tested
- umbilical cord blood mononuclear stem cells
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
umbilical cord blood mononuclear stem cellsOther
UCB samples (70-130 ml of initial volume) were aseptically drained in blood-collection bags with 35 mL of citrate phosphate dextrose (CPDA-1) anticoagulant. Red blood cell-depleted/plasma-reduced nucleated cells were isolated by sedimentation in dextran-40, resuspended in autologous plasma with 10% dimethylsulfoxide and 1% dextran-40, and aliquoted in 3.8 ml vials with target doses of 3.0±0.2 x 10⁶ cells per vial. Vials were cooled to -90⁰C, quarantined in the vapor phase of liquid nitrogen. Tested for HIV-1/2, hepatitis B and C, HTLV-1/2, HSV-1 and -2, CMV, syphilis, and sterility. Characterized by AB0/Rh, CD34/CD45-positive cell content. Before injection sample thaws in the water bath at +37⁰C and washes from an excess amount of DMSO in 40 mL of physiological saline containing 2.5% human serum albumin and 5% dextran-40. After centrifugation and supernatant withdrawal, the UCB resuspends in 40 ml of the same solution at a target dose of 250±10 x 10⁶ viable cells per sample.
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | Patients receive group and rhesus compatible UCB cells through intravenous bolus injections (4 injections at 2-week intervals) after pre-medication with Clemastine (0.025 mg/kg body weight, intravenously). One dose is 250±10 x 10⁶ viable cells per sample. Receives: Other: umbilical cord blood mononuclear stem cells |
| Control | No intervention | Patients receives standard therapy (applied behavioral analysis, speech therapy). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment Emergent Adverse Events
Assessed by Adverse Event Grading Scale. It categorized adverse events according to severity.
Mild Adverse Event - event results in mild or transient discomfort, not requiring intervention or treatment; does not limit or interfere with daily activities (e.g., insomnia, mild headache).
Moderate Adverse Event - event is sufficiently discomforting so as to limit or interfere with daily activities; may require interventional treatment (e.g., fever requiring antipyretic medication).
Severe and undesirable Adverse Event - event results in significant symptoms that prevent normal daily activities; may require hospitalization or invasive intervention (e.g., anemia resulting in blood transfusion).
Measured: 24 hours after infusion
Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Checklist for Autism Spectrum Disorders (CASD)
The CASD rates 30 symptoms of autism, covering six domains: problems with social interaction, perseveration, somatosensory disturbance, atypical communication and development, mood, problems with attention and safety. Symptoms are scored as present or absent based on information drawn from various sources, including teacher, caregiver, parent, observation of the child, and available records. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 30 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.
Measured: at baseline, 3d and 6th month
Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Autism Treatment Evaluation Checklist (ATEC)
The purpose of the ATEC is to measure a change in a patient due to various interventions. It consists of four subtests: Speech/Language Communication (14 items); Sociability (20 items); Sensory/ Cognitive Awareness (18 items); Health/Physical/Behavior (25 items). The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.
Measured: at baseline, 3d and 6th month
Secondary outcomes (4)
"Digit Span" subscale of Wechsler Intelligence Scale for Children (WISC)
It assesses short-term auditory memory, concentration. The child is asked to repeat dictated series of digits forwards and other series backwards. Series begin with two digits and keep increasing in length, with two trials at each length. A primary score ranges from 0 to 9 points for forward series and from 0 to 8 points for backward series. The primary score is further converted into a final score in accordance with the age of the patient. A higher score correlates with better cognitive functioning.
Measured: at baseline, 6th month
"Picture completion" subscale of Wechsler Intelligence Scale for Children (WISC)
This subscale measures alertness to detail, visual discrimination. The child is asked to identify missing parts of pictures. A primary score ranges from 0 to 20 points. The primary score is further converted into a final score in accordance with the age of the patient. A higher score means better cognitive functioning.
Measured: at baseline, 6th month
"Block design" subscale of Wechsler Intelligence Scale for Children (WISC)
It helps to measure spatial analysis, abstract visual problem-solving. It provides blocks and pictures, and the child must put the blocks together to re-create what's in the picture of the blocks. A primary score ranges from 0 to 55 points. The primary score is further converted into a final score in accordance with the age of the patient. A higher score means better cognitive functioning.
Measured: at baseline, 6th month
"Coding" subscale of Wechsler Intelligence Scale for Children (WISC)
It assesses visual-motor coordination, speed, concentration. Coding A is used for children under age 8. The child is asked to mark rows of shapes with different lines according to a code as quickly as possible for 2 minutes. A primary score ranges (Coding A) from 0 to 50 points. Coding B is used for children aged 8 and older. The child is asked to transcribe a digit-symbol code as quickly as possible for two minutes. A primary score ranges (Coding B) from 0 to 93 points. The primary score is further converted into the final score in accordance with the age of the patient. A higher score means better cognitive functioning.
Measured: at baseline, 6th month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2021
- Study start4 Nov 2019
- Primary completion31 Jan 2021
- Study completion31 Jul 2021
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Petersburg Bekhterev Research Psychoneurological Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04710810
- Sponsor's protocol ID
- SCA1512
- Status last verified
- Aug 2021
- First submitted
- 28 Dec 2020
- Last updated
- 4 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transplantation of Human Allogenic Cord Blood Mononuclear Stem Cells in Autism: Safety and Efficacy of the Method
- ClinicalTrials.gov ID
- NCT04710810
- Acronym / sponsor ID
- SCA1512
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- St. Petersburg Bekhterev Research Psychoneurological InstituteUniversity, hospital or other
- Study start
- 4 Nov 2019 (actual)
- Primary completion
- 31 Jan 2021 (actual)
- Study completion
- 31 Jul 2021 (actual)
- First posted
- 15 Jan 2021 (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.
Purpose. The goals of the study are to assess the safety of the intravenous infusion of umbilical cord blood (UCB) cells in patients with autism and to confirm changes in social/ communicative skills and cognitive functioning after four infusions of ABO/Rh-matched UCB stem cells.
Material and methods. The sample comprises 30 patients (27 males, 3 females) aged between 3 and 11 years with ASD under the care of the National Medical Research Center for Psychiatry and Neurology (Saint-Petersburg, Russia). Participants are randomly assigned to either the control group (14 males, 1 female) or the experimental group (13 males, 2 females). The experimental group receives intravenous injections of UCB cells four times with a two-week gap between injections. The control group receives standard therapy.
The dynamic of cognitive functions and social/communicative skills assess with Checklist for autism spectrum disorders (CASD), Autism treatment evaluation checklist (ATEC), subscales of Wechsler Intelligence Scale for Children (WISC) - "Digit Span", "Picture completion", "Block design", "Coding".
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Other: umbilical cord blood mononuclear stem cellsPatients receive group and rhesus compatible UCB cells through intravenous bolus injections (4 injections at 2-week intervals) after pre-medication with Clemastine (0.025 mg/kg body weight, intravenously). One dose is 250±10 x 10⁶ viable cells per sample. |
| Control | No intervention | –Patients receives standard therapy (applied behavioral analysis, speech therapy). |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- diagnosed ASD by ICD-10 criteria
Key exclusion criteria (4 listed)
- acute somatic diseases
- indication for surgical (neurosurgical) therapy
- acute or chronic infectious diseases
- history of acute allergic reactions
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 |
|---|---|---|
| umbilical cord blood mononuclear stem cellsUCB samples (70-130 ml of initial volume) were aseptically drained in blood-collection bags with 35 mL of citrate phosphate dextrose (CPDA-1) anticoagulant. Red blood cell-depleted/plasma-reduced nucleated cells were isolated by sedimentation in dextran-40, resuspended in autologous plasma with 10% dimethylsulfoxide and 1% dextran-40, and aliquoted in 3.8 ml vials with target doses of 3.0±0.2 x 10⁶ cells per vial. Vials were cooled to -90⁰C, quarantined in the vapor phase of liquid nitrogen. Tested for HIV-1/2, hepatitis B and C, HTLV-1/2, HSV-1 and -2, CMV, syphilis, and sterility. Characterized by AB0/Rh, CD34/CD45-positive cell content. Before injection sample thaws in the water bath at +37⁰C and washes from an excess amount of DMSO in 40 mL of physiological saline containing 2.5% human serum albumin and 5% dextran-40. After centrifugation and supernatant withdrawal, the UCB resuspends in 40 ml of the same solution at a target dose of 250±10 x 10⁶ viable cells per sample. | Other | Experimental |
Primary outcome
Incidence of Treatment Emergent Adverse Events
Time frame: 24 hours after infusion
Assessed by Adverse Event Grading Scale. It categorized adverse events according to severity. Mild Adverse Event - event results in mild or transient discomfort, not requiring intervention or treatment; does not limit or interfere with daily activities (e.g., insomnia, mild headache). Moderate Adverse Event - event is sufficiently discomforting so as to limit or interfere with daily activities; may require interventional treatment (e.g., fever requiring antipyretic medication). Severe and undesirable Adverse Event - event results in significant symptoms that prevent normal daily activities; may require hospitalization or invasive intervention (e.g., anemia resulting in blood transfusion).
Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Checklist for Autism Spectrum Disorders (CASD)
Time frame: at baseline, 3d and 6th month
The CASD rates 30 symptoms of autism, covering six domains: problems with social interaction, perseveration, somatosensory disturbance, atypical communication and development, mood, problems with attention and safety. Symptoms are scored as present or absent based on information drawn from various sources, including teacher, caregiver, parent, observation of the child, and available records. The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 30 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.
Changes in severity of autistic symptoms from baseline at 3d and 6th month (measured by Autism Treatment Evaluation Checklist (ATEC)
Time frame: at baseline, 3d and 6th month
The purpose of the ATEC is to measure a change in a patient due to various interventions. It consists of four subtests: Speech/Language Communication (14 items); Sociability (20 items); Sensory/ Cognitive Awareness (18 items); Health/Physical/Behavior (25 items). The scores from each subscale are combined in order to calculate a Total Score, which ranges from 0 to 179 points. A lower score indicates less severe symptoms of ASD and a higher score correlates with more severe symptoms of ASD.
4 secondary outcomes
"Digit Span" subscale of Wechsler Intelligence Scale for Children (WISC)
Time frame: at baseline, 6th month
"Picture completion" subscale of Wechsler Intelligence Scale for Children (WISC)
Time frame: at baseline, 6th month
"Block design" subscale of Wechsler Intelligence Scale for Children (WISC)
Time frame: at baseline, 6th month
"Coding" subscale of Wechsler Intelligence Scale for Children (WISC)
Time frame: at baseline, 6th month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2021.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing umbilical cord blood mononuclear stem cells in people with Autism Spectrum Disorder, run by St. Petersburg Bekhterev Research Psychoneurological Institute 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 1 inclusion and 4 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 4 Aug 2021. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of Treatment Emergent Adverse Events (time frame: 24 hours after infusion). 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 4 Aug 2021.