Autism Spectrum DisorderPhase 1NCT02176317
Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)
A Phase 1 study testing Autologous Umbilical Cord Blood in people with Autism Spectrum Disorder, run by Duke University at 1 site in United States.
Trial summary
This study is a prospective phase 1 single-center trial designed to determine the safety of a single intravenous infusion of autologous umbilical cord blood in children with Autism Spectrum Disorder (ASD) and assess the feasibility of various outcome measures to determine which measure(s) can be used as primary and… Read more about this study
- Who is it for?
- Ages 24 to 72 months
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- Autologous Umbilical Cord Blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2014to Dec 2015
- 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 24 to 72 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 24 months to ≤72months at the time of visit 1
- Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using all three of the following measures:
- Autism Diagnostic Observation Schedule - Toddler or Generic (ADOS)
- Autism Diagnostic Interview-Revised (ADI-R)
- DSM-5 checklist
- IQ ≥ 35 on Stanford Binet Intelligence Scale or similar standardized test
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- Autologous umbilical cord blood available from a cord blood bank with a minimum total nucleated cell dose of ≥ 1 x 107 cells/kilogram of subject weight that meets acceptance criteria outlined in section 6.0 with confirmed HLA matching
- Stable on current medications for at least 2 months prior to infusion of cord blood
- Ability to travel to Duke University three times (0, 6, 12 mo.), parent/guardian able to participate in electronic communication tracking two times in the study and interim phone surveys every 3 months
- Parental consent
- Subject and parent/guardian must be English speaking
Who cannot join
- Unwilling to commit to follow up for a year
- History of prior cell therapy
- Use of IVIG or other anti-inflammatory medications with the exception of NSAIDs
- Medical records indicate that child has genetic or other syndromes such as fragile X, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cerebral palsy, cystic fibrosis, muscular dystrophy, Crohn's disease, or rheumatoid disease
- Co-morbid condition that would influence child's performance on assessments.
- Central Nervous System (CNS) infection
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- History of unstable epilepsy or uncontrolled seizure disorder, infantile spasms, Lennox Gastaut syndrome, Dravet syndrome
- Known pathogenic copy number variation (CNV) (e.g. 16p11.2, 15q13.2, 2q13.3)
- Significant sensory (i.e., deafness, blind) or motor impairment (CP) (if using Language Environment Analysis (LENA), no uncorrected hearing impairment)
- Presence of obvious physical dysmorphology
- Review of medical records indicates ASD diagnosis not likely or other serious complicating genetic or medical condition present
- Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin>1.3mg/dL
- Clinically significant abnormalities in Complete Blood Count (CBC): Hemoglobin < 10.0 g/dL, White Blood Count (WBC) < 3.8 x 10e9, Platelets < 150x 10e9.
- Known metabolic disorder, mitochondrial dysfunction
- Uncontrolled infection, presence of or infection with HIV
- Active malignancy
- Macroencephaly or microencephaly ( >2 standard deviations in the relevant direction between head circumference and height)
- Change in current stable use of psychoactive medications; as per parent report.
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27705
About this study
This study is a prospective phase 1 single-center trial designed to determine the safety of a single intravenous infusion of autologous umbilical cord blood in children with Autism Spectrum Disorder (ASD) and assess the feasibility of various outcome measures to determine which measure(s) can be used as primary and secondary endpoints for a future randomized phase 2 clinical trial. All subjects will receive infusion of cord blood cells at baseline with follow up assessments at 6 and 12 months.
Read the detailed description
Autism Spectrum Disorder (ASD) is a neurodevelopment disorder with early onset in life. Currently, available treatments for patients with ASD are supportive, but not curative. Umbilical cord blood (UCB) has been shown to lessen the clinical and radiographic impact of hypoxic brain injury and stroke in animal models and in infants with hypoxic ischemic encephalopathy. UCB also engrafts and differentiates in the brain, facilitating neural cell repair in animal models and human patients with inborn errors of metabolism undergoing allogeneic, unrelated donor UCB transplantation. Infusion of autologous UCB does not require immunosuppression and has been shown to be safe in young children with brain injuries such as cerebral palsy and stroke. In this study, the investigators hypothesize that infusion of a patient's own umbilical cord blood cells (UCB) can offer neural protection/repair in the brain and reduction of inflammation associated with this disorder.
- Conditions
- Autism Spectrum Disorder
- Being tested
- Autologous Umbilical Cord Blood
- Keywords
- Autism Spectrum Disorder, ASD, Umbilical cord blood, Autologous cord blood, cord blood
- 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.
Autologous Umbilical Cord BloodBiological
All participants will receive autologous umbilical cord blood cells with a pre-cryopreservation cell dose of 1-5 x 10^7 Total Nucleated Cells (TNC)/kilogram of subject body weight. The cells will be administered as a single intravenous (into the vein) infusion over 2 to 25 minutes
| Study group | Role | What they receive |
|---|---|---|
| Autologous Umbilical Cord Blood (UCB) | Experimental | All participants will receive a single intravenous (into the vein) infusion of autologous umbilical cord blood cells. Receives: Biological: Autologous Umbilical Cord Blood |
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.
Measured: up to 12 months
Secondary outcomes (1)
The primary efficacy measure will be change in the Vineland Adaptive Behavior Scale- II
Measured: up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2014 (estimated)
- Study startJun 2014
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation, PerkinElmer, Inc.
- Responsible party
- Principal investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Joanne Kurtzberg, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02176317
- Sponsor's protocol ID
- Pro00052449
- Status last verified
- Dec 2019
- First submitted
- 16 Jun 2014
- Last updated
- 3 Dec 2019
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Simhal AK, Carpenter KLH, Nadeem S, Kurtzberg J, Song A, Tannenbaum A, Sapiro G, Dawson G. Measuring robustness of brain networks in autism spectrum disorder with Ricci curvature. Sci Rep. 2020 Jul 2;10(1):10819. doi: 10.1038/s41598-020-67474-9.
PubMed 32616759
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)
- ClinicalTrials.gov ID
- NCT02176317
- Acronym / sponsor ID
- Pro00052449
- Conditions
- Autism Spectrum Disorder
- Keywords
- Autism Spectrum Disorder, ASD, Umbilical cord blood, Autologous cord blood, cord blood
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- The Marcus Foundation, PerkinElmer, Inc.
- Study start
- Jun 2014
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 27 Jun 2014 (estimated)
- 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.
This study is a prospective phase 1 single-center trial designed to determine the safety of a single intravenous infusion of autologous umbilical cord blood in children with Autism Spectrum Disorder (ASD) and assess the feasibility of various outcome measures to determine which measure(s) can be used as primary and secondary endpoints for a future randomized phase 2 clinical trial. All subjects will receive infusion of cord blood cells at baseline with follow up assessments at 6 and 12 months.
Detailed description
Autism Spectrum Disorder (ASD) is a neurodevelopment disorder with early onset in life. Currently, available treatments for patients with ASD are supportive, but not curative. Umbilical cord blood (UCB) has been shown to lessen the clinical and radiographic impact of hypoxic brain injury and stroke in animal models and in infants with hypoxic ischemic encephalopathy. UCB also engrafts and differentiates in the brain, facilitating neural cell repair in animal models and human patients with inborn errors of metabolism undergoing allogeneic, unrelated donor UCB transplantation. Infusion of autologous UCB does not require immunosuppression and has been shown to be safe in young children with brain injuries such as cerebral palsy and stroke. In this study, the investigators hypothesize that infusion of a patient's own umbilical cord blood cells (UCB) can offer neural protection/repair in the brain and reduction of inflammation associated with this disorder.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Umbilical Cord Blood (UCB) | Experimental | Biological: Autologous Umbilical Cord BloodAll participants will receive a single intravenous (into the vein) infusion of autologous umbilical cord blood cells. |
Who may qualify
- Ages
- 24 to 72 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Age ≥ 24 months to ≤72months at the time of visit 1
- Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using all three of the following measures:
- IQ ≥ 35 on Stanford Binet Intelligence Scale or similar standardized test
- Autologous umbilical cord blood available from a cord blood bank with a minimum total nucleated cell dose of ≥ 1 x 107 cells/kilogram of subject weight that meets acceptance criteria outlined in section 6.0 with confirmed HLA matching
- Stable on current medications for at least 2 months prior to infusion of cord blood
Key exclusion criteria (18 listed)
- Unwilling to commit to follow up for a year
- History of prior cell therapy
- Use of IVIG or other anti-inflammatory medications with the exception of NSAIDs
- Medical records indicate that child has genetic or other syndromes such as fragile X, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cerebral palsy, cystic fibrosis, muscular dystrophy, Crohn's disease, or rheumatoid disease
- Co-morbid condition that would influence child's performance on assessments.
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 Umbilical Cord BloodAll participants will receive autologous umbilical cord blood cells with a pre-cryopreservation cell dose of 1-5 x 10^7 Total Nucleated Cells (TNC)/kilogram of subject body weight. The cells will be administered as a single intravenous (into the vein) infusion over 2 to 25 minutes | Biological | Autologous Umbilical Cord Blood (UCB) |
Primary outcome
Number of participants with non-serious and serious adverse events.
Time frame: up to 12 months
1 secondary outcome
The primary efficacy measure will be change in the Vineland Adaptive Behavior Scale- II
Time frame: up to 12 months
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 3 Dec 2019.
- Derived from this study: PubMed 32616759Simhal AK, Carpenter KLH, Nadeem S, Kurtzberg J, Song A, Tannenbaum A, Sapiro G, Dawson G. Measuring robustness of brain networks in autism spectrum disorder with Ricci curvature. Sci Rep. 2020 Jul 2;10(1):10819. doi: 10.1038/s41598-020-67474-9.
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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.
Joanne Kurtzberg, MD principal investigator
Duke University
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 Autologous Umbilical Cord Blood in people with Autism Spectrum Disorder, run by Duke University 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 11 inclusion and 18 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 3 Dec 2019. 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. 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?
Number of participants with non-serious and serious adverse events. (time frame: up to 12 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 NCT02176317, 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 3 Dec 2019.