AutismPhase 2NCT01638819
Autologous Cord Blood Stem Cells for Autism
A Phase 2 study testing Autologous Cord Blood Stem Cells in people with Autism, run by Sutter Health at 1 site in United States.
Trial summary
Evaluate the efficacy of one infusion of stem cells from autologous umbilical cord blood in patients with autism over six months after infusion as measured by changes in expressive and receptive language. Read more about this study
- Who is it for?
- Ages 2 to 7 years
- Conditions the study is for
- Autism
- What is being tested?
- Autologous Cord Blood Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2012to Dec 2016
- Phase
- Phase 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 2 to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 2 to 7 years of age
- Diagnosis of Autistic Disorder as diagnosed by the Diagnostic and Statistical Manual, 4th Edition, Text Revision (DSM-IV-TR) developmental delays, and ADOS
- A sufficient quantity of autologous cord blood stored at Cord Blood Registry that was stored and processed using the Thermogenesis AutoXpress Platform
- Stable on any current medications for at least 2 months prior to infusion of cord blood
- Medical records indicating that patient does not have genetic conditions such as cerebral palsy, cystic fibrosis, muscular dystrophy, crohns disease, rheumatoid disease, fragile X, Retts Syndrome, Angelman Syndrome, tuberous sclerosis, epilepsy, or known genetic defects that overlap autism spectrum.
- Results of an EEG within 12-months of baseline
- English speaking
Who cannot join
- CNS infection
- Extreme prematurity (< 34 weeks gestation)
- Severe Cognitive Disability IQ below 45 with autism
- Clinical seizure activity within 6 months of baseline
- Lennox Gastaut syndrome or infantile spasms
- Dravet syndrome
Show all 24Show fewer
- HIV, renal or hepatic impairment
- Prior hematological or malignant disease
- Fever of 101 F within 2 weeks prior to infusion
- Serious CNS infection or trauma
- Unwilling to commit to follow-up
- Mental illness including schizophrenia
- Pervasive Developmental Disorder-Not Otherwise Specified
- Asperger's Disorder
- Cord blood unit is less than 85% viable, has a TNC of less than 10 million/kg, or sterility testing results are positive
- Garlic allergy
- Previous adverse reaction to Dimethyl Sulfoxide (DMSO)
- Maternal medical records indicate communicable diseases including HIV, Hepatitis B or C, syphilis, cytomegalovirus (CMV)
- Currently taking anti-inflammatory medications
- History of asthma who may potentially require treatment with steroids
- Inflammatory Disease
- Renal/hepatic disease: serum Creatinine > 1.5 mg/dl and total Bilirubin > 1.5 mg/dl
- Allergic to diphenhydramine (Benadryl)
- Treatment with chelation therapy, hyperbaric oxygen therapy, pig worm therapy, or other alternative therapies the investigator deems clinically relevant
Trial locations and contacts
1 site in 1 country.
United States1 site
Sutter Pediatric Neurology
Sacramento, California, 95816
About this study
Official title: A Randomized, Blinded, Placebo-controlled, Crossover Study to Assess the Efficacy of Stem Cells From Autologous Umbilical Cord Blood to Improve Language and Behavior in Children With Autism
Evaluate the efficacy of one infusion of stem cells from autologous umbilical cord blood in patients with autism over six months after infusion as measured by changes in expressive and receptive language.
Also demonstrate improved behavior, learning, and changes in Serum tumor necrosis factor alpha (TNF-α), tumor necrosis factor beta (TNF-β), interleukin 1-alpha (IL-1α), interleukin 1-beta (IL-1β), interleukin 6 (IL-6), interleukin 10 (IL-10), and interleukin 13 (IL-13).
Read the detailed description
This is a single-center, randomized, placebo-controlled, crossover outpatient study with 15 subjects receiving one infusion of autologous umbilical cord blood (AUCB) containing a minimum of 10 million total nucleated cells per kilogram (TNC/kg) and 15 subjects receiving an infusion of placebo (saline). After the 24-week follow-up testing is conducted, the groups will crossover so that patients who initially received AUCB will receive placebo and patients who received placebo at baseline will receive the cord blood. Both groups will be tested again 24-weeks after infusion. The neuropsychologist, PI, staff from Cord Blood Registry (CBR), and parents will be blinded as to the infusion sequence.
The duration of participation for each study subject is approximately 55 weeks. This includes one screening visit over a period of approximately 6 weeks, one visit for baseline testing, one day for infusion of TNC (minimum 10 million/kg) or saline placebo followed by 24 weeks of follow-up. A second baseline visit is conducted at week-24 with the second infusion of TNC or saline placebo occurring 5-7 days after. Twenty-four additional weeks of follow-up occur after the second infusion.
- Conditions
- Autism
- Being tested
- Autologous Cord Blood Stem Cells, Placebo
- Medical subject headings
- Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Cord Blood Stem CellsBiological
One infusion of 60 ml syringe of study product
PlaceboBiological
Saline
| Study group | Role | What they receive |
|---|---|---|
| Autologous Cord Blood Stem Cells | Experimental | Not described Receives: Biological: Autologous Cord Blood Stem Cells |
| Placebo | Placebo comparator | Saline Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Language (Total Standard Score, Range 40 - 160)
Change in language as measured by the Receptive One-Word Vocabulary Test (ROWVT-4) and Expressive One-Word Vocabulary Test (EOWVT-4) at baseline and six months following infusion of stem cells from AUCB or infusion of placebo.
The ROWPVT-4 and EOWPVT-4 were administered by a neuropsychologist and are norm-referenced assessments. The ROWPVT-4 tests an individual's ability to match a spoken word with an image of an object, action, or concept. The EOWPVT-4 tests an individual's ability to name, with one word, objects, actions, and concepts when presented with color illustrations. The tests target the ability to understand the meaning of words spoken and name what is depicted on a test plate without context.
Scores of 85-115 are considered to be within the average range of functioning.
Measured: Baseline and 6 months
Secondary outcomes (2)
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Change in the Vineland Adaptive Behavior and Socialization Scales (2nd edition) between baseline and six months after infusion of AUCB containing stem cells.
Vineland Adaptive Behavior and Socialization Scales consist of the following subparts: Daily Living Skills, Socialization, and Adaptive Behavior Composite (ABC). These are questionnaires completed by a parent or caregiver. Scores above 80 are classified using approximately the same ranges as IQ tests. Scores below 80 are categorized as borderline adaptive functioning (70-80); mildly deficient adaptive functioning (51-69); moderately deficient adaptive behavior (36-50); severely deficient adaptive behavior; (20-35); and markedly or profoundly deficient adaptive behavior (<20).
Measured: Baseline and 6 months
Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
The Stanford Binet, version 5, was used to assess the brain function. It can assess the level of intelligence across several age spans and ability levels. The Stanford-Binet looks at intelligence in five areas. In this study 2 areas were looked at: Knowledge and Fluid Reasoning that were age and condition appropriate. Each sub-test has a mean of 10 and a standard deviation of 3. The standard deviation indicates how far above or below the norm the subject's score is. Scores of 7 to 13 are considered to be within the average range of functioning.
Measured: Baseline and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2012 (estimated)
- Study startAug 2012
- Primary completionDec 2015
- Study completionDec 2016
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AutismStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sutter Health
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Michael Chez, MD, Sutter Health
- Investigators
- Michael Chez, MD, Principal investigator, Sutter Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01638819
- Sponsor's protocol ID
- CB2011Chez
- Status last verified
- Aug 2018
- First submitted
- 26 Jun 2012
- Last updated
- 20 Aug 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Mauron A. [Autism, stem cells, and magical powder]. Rev Med Suisse. 2012 Sep 19;8(354):1795. No abstract available. French.
PubMed 23097920
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Blinded, Placebo-controlled, Crossover Study to Assess the Efficacy of Stem Cells From Autologous Umbilical Cord Blood to Improve Language and Behavior in Children With Autism
- ClinicalTrials.gov ID
- NCT01638819
- Acronym / sponsor ID
- CB2011Chez
- Conditions
- Autism
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Sutter HealthUniversity, hospital or other
- Study start
- Aug 2012
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 12 Jul 2012 (estimated)
- Results
- Posted 20 Aug 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluate the efficacy of one infusion of stem cells from autologous umbilical cord blood in patients with autism over six months after infusion as measured by changes in expressive and receptive language.
Also demonstrate improved behavior, learning, and changes in Serum tumor necrosis factor alpha (TNF-α), tumor necrosis factor beta (TNF-β), interleukin 1-alpha (IL-1α), interleukin 1-beta (IL-1β), interleukin 6 (IL-6), interleukin 10 (IL-10), and interleukin 13 (IL-13).
Detailed description
This is a single-center, randomized, placebo-controlled, crossover outpatient study with 15 subjects receiving one infusion of autologous umbilical cord blood (AUCB) containing a minimum of 10 million total nucleated cells per kilogram (TNC/kg) and 15 subjects receiving an infusion of placebo (saline). After the 24-week follow-up testing is conducted, the groups will crossover so that patients who initially received AUCB will receive placebo and patients who received placebo at baseline will receive the cord blood. Both groups will be tested again 24-weeks after infusion. The neuropsychologist, PI, staff from Cord Blood Registry (CBR), and parents will be blinded as to the infusion sequence.
The duration of participation for each study subject is approximately 55 weeks. This includes one screening visit over a period of approximately 6 weeks, one visit for baseline testing, one day for infusion of TNC (minimum 10 million/kg) or saline placebo followed by 24 weeks of follow-up. A second baseline visit is conducted at week-24 with the second infusion of TNC or saline placebo occurring 5-7 days after. Twenty-four additional weeks of follow-up occur after the second infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Autologous Cord Blood Stem Cells | Experimental | Biological: Autologous Cord Blood Stem Cells |
| Placebo | Placebo comparator | Biological: PlaceboSaline |
Who may qualify
- Ages
- 2 to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Age 2 to 7 years of age
- Diagnosis of Autistic Disorder as diagnosed by the Diagnostic and Statistical Manual, 4th Edition, Text Revision (DSM-IV-TR) developmental delays, and ADOS
- A sufficient quantity of autologous cord blood stored at Cord Blood Registry that was stored and processed using the Thermogenesis AutoXpress Platform
- Stable on any current medications for at least 2 months prior to infusion of cord blood
- Medical records indicating that patient does not have genetic conditions such as cerebral palsy, cystic fibrosis, muscular dystrophy, crohns disease, rheumatoid disease, fragile X, Retts Syndrome, Angelman Syndrome, tuberous sclerosis, epilepsy, or known genetic defects that overlap autism spectrum.
Key exclusion criteria (24 listed)
- CNS infection
- Extreme prematurity (< 34 weeks gestation)
- Severe Cognitive Disability IQ below 45 with autism
- Clinical seizure activity within 6 months of baseline
- Lennox Gastaut syndrome or infantile spasms
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 |
|---|---|---|
| Autologous Cord Blood Stem CellsOne infusion of 60 ml syringe of study product | Biological | Autologous Cord Blood Stem Cells |
| PlaceboSaline | Biological | Placebo |
Primary outcome
Change in Language (Total Standard Score, Range 40 - 160)
Time frame: Baseline and 6 months
Change in language as measured by the Receptive One-Word Vocabulary Test (ROWVT-4) and Expressive One-Word Vocabulary Test (EOWVT-4) at baseline and six months following infusion of stem cells from AUCB or infusion of placebo. The ROWPVT-4 and EOWPVT-4 were administered by a neuropsychologist and are norm-referenced assessments. The ROWPVT-4 tests an individual's ability to match a spoken word with an image of an object, action, or concept. The EOWPVT-4 tests an individual's ability to name, with one word, objects, actions, and concepts when presented with color illustrations. The tests target the ability to understand the meaning of words spoken and name what is depicted on a test plate without context. Scores of 85-115 are considered to be within the average range of functioning.
2 secondary outcomes
Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)
Time frame: Baseline and 6 months
Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)
Time frame: Baseline and 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 23097920Mauron A. [Autism, stem cells, and magical powder]. Rev Med Suisse. 2012 Sep 19;8(354):1795. No abstract available. French.
- Sutter Institute for Medical ResearchLink provided by the sponsor.
Related trials for Autism
RecruitingPhase 1
Cannabidiol (CBD) in Adults With ASD
Autism Spectrum Disorder
RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride for the Treatment of Psychiatric Disorders in Children and Adolescents
Schizophrenia, Autism-Related Irritability
Not yet recruiting
Sensory-integration Therapy Verses Augumented Reality-based Training in ASD
Autism Spectrum Disorder
Recruiting
Cumulative and Booster Effects of Multisession Prefrontal tDCS in ASD Adolescents
Transcranial Direct Current Stimulation, Autistic Spectrum Disorder
Not yet recruiting
"Family Resilience And Mind-body Enhancement" (FRAME): A Randomised Controlled Trial
Autism
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.
Michael Chez, MD principal investigator
Sutter Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous Cord Blood Stem Cells in people with Autism, run by Sutter Health at 1 site in United States.
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 7 inclusion and 24 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 20 Aug 2018. 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 United States. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 20 Aug 2018). 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?
Change in Language (Total Standard Score, Range 40 - 160) (time frame: Baseline and 6 months). 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 NCT01638819, which also lists 1 linked publication.
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 20 Aug 2018.