Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)
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ClinicalTrials.gov Identifier: NCT02176317 |
Recruitment Status :
Completed
First Posted : June 27, 2014
Last Update Posted : December 3, 2019
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Sponsor:
Duke University
Collaborators:
The Marcus Foundation
PerkinElmer, Inc.
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University
Tracking Information | ||||
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First Submitted Date ICMJE | June 16, 2014 | |||
First Posted Date ICMJE | June 27, 2014 | |||
Last Update Posted Date | December 3, 2019 | |||
Study Start Date ICMJE | June 2014 | |||
Actual Primary Completion Date | December 2015 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Number of participants with non-serious and serious adverse events. [ Time Frame: up to 12 months ] | |||
Original Primary Outcome Measures ICMJE | Same as current | |||
Change History | ||||
Current Secondary Outcome Measures ICMJE |
The primary efficacy measure will be change in the Vineland Adaptive Behavior Scale- II [ Time Frame: up to 12 months ] | |||
Original Secondary Outcome Measures ICMJE | Same as current | |||
Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Same as current | |||
Descriptive Information | ||||
Brief Title ICMJE | Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD) | |||
Official Title ICMJE | Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD) | |||
Brief 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 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 Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 1 | |||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Autism Spectrum Disorder | |||
Intervention ICMJE | Biological: Autologous Umbilical Cord Blood
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
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Study Arms ICMJE | Experimental: Autologous Umbilical Cord Blood (UCB)
All participants will receive a single intravenous (into the vein) infusion of autologous umbilical cord blood cells.
Intervention: Biological: Autologous Umbilical Cord Blood
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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. | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
25 | |||
Original Estimated Enrollment ICMJE |
20 | |||
Actual Study Completion Date ICMJE | December 2015 | |||
Actual Primary Completion Date | December 2015 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 24 Months to 72 Months (Child) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT02176317 | |||
Other Study ID Numbers ICMJE | Pro00052449 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Current Responsible Party | Joanne Kurtzberg, MD, Duke University | |||
Original Responsible Party | Same as current | |||
Current Study Sponsor ICMJE | Duke University | |||
Original Study Sponsor ICMJE | Same as current | |||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | Duke University | |||
Verification Date | December 2019 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |