Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism

• ClinicalTrials.gov Identifier: NCT02192749
• Recruitment Status: Completed
• First Posted: July 17, 2014
• Last Update Posted: October 13, 2017
• Sponsor: Translational Biosciences
• Information provided by (Responsible Party): Translational Biosciences

Tracking Information

• First Submitted Date: July 12, 2014
• First Posted Date: July 17, 2014
• Last Update Posted Date: October 13, 2017
• Study Start Date: July 2014
• Actual Primary Completion Date: August 2017 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: July 15, 2014): Number of participants with adverse events [ Time Frame: 89 weeks ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 15, 2014)

• Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC) [ Time Frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks ]
• Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS) [ Time Frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks ]
• Change from baseline macrophage-derived chemokine (MDC) [ Time Frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks ]
• Change from baseline thymus and activation-regulated chemokine (TARC) [ Time Frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
• Official Title: Open, Prospective Trial of Treatment of Autism Spectrum Disorders (ASD) Using Intravenous Infusion of Umbilical Cord Tissue Mesenchymal Stem Cells (UC-MSC)
• Brief Summary: Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is safe and will induce a therapeutic effect in autism patients.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Autism
• Intervention: Biological: Umbilical cord mesenchymal stem cells

Study Arms

Experimental: Umbilical cord mesenchymal stem cells

Intervention: Biological: Umbilical cord mesenchymal stem cells

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 15, 2014): 20
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: August 2017
• Actual Primary Completion Date: August 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Male or Female
• Ages 6 to 16
• Diagnostic and Statistical Manual of Mental Disorders (DSM IV) diagnosis of autism confirmed by Autism Diagnostic Observation Schedule (ADOS) and/or Autism Diagnostic Interview-Revised (ADI-R)
• No anticipated changes in treatment for the study duration (e.g., diet, nutrients)
• No additional biomedical treatments started 6 weeks prior to enrollment
• No changes in dietary management for 3 months prior to enrollment
• Ambulatory or require minimum support walking, per parent
• Able to sit still for 5 minutes or longer with a preferred toy item, per parent
• Adequate vision and hearing for the purposes of test administration, per parent
• Adequate arm-hand-finger coordination (i.e., able to point) for learning and cognitive tasks used in outcome measurement, per parent
• Stable and controlled mental disorder
• Under the care of a caregiver willing to participate by attending regularly scheduled appointments and completing the necessary measures
• Normal heavy metals test for lead and mercury levels performed within 30 days of first stem cell infusion
• Must provide name and specialty of specialist who has made Autism Spectrum Disorder (ASD) diagnosis
• Adequate financial means to cover $7,200 (US Dollars) plus travel expenses

Exclusion Criteria:

• Significant prematurity at birth (less than 32 weeks gestation); or birth weight significantly below normal for gestational age (SGA - small for gestational age)
• mental retardation
• seizure disorder
• auto-immune conditions
• history of head trauma and other neurological or medical conditions
• Abnormal heavy metals test for lead and mercury performed within 30 days of first stem cell infusion
• Prior stem cell therapy of any kind

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Years to 16 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Panama

Administrative Information

• NCT Number: NCT02192749
• Other Study ID Numbers: TBS-UCMSC-ASD001
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Translational Biosciences
• Original Responsible Party: Same as current
• Current Study Sponsor: Translational Biosciences
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Nelson Novarro, MD
• Principal Investigator: Jorge Paz-Rodriguez, MD; Translational Biosciences / Stem Cell Institute Panama

• PRS Account: Translational Biosciences
• Verification Date: October 2017