Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02192749
• Date of registration: 12/07/2014
• Prospective Registration: No
• Primary sponsor: Translational Biosciences
• Public title: Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
• Scientific title: Open, Prospective Trial of Treatment of Autism Spectrum Disorders (ASD) Using Intravenous Infusion of Umbilical Cord Tissue Mesenchymal Stem Cells (UC-MSC)
• Date of first enrolment: July 2014
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02192749
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Panama

Contacts

• Name: Nelson Novarro, MD

• Name: Jorge Paz-Rodriguez, MD
• Affiliation: Translational Biosciences / Stem Cell Institute Panama

Key inclusion & exclusion 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



Age minimum: 6 Years
Age maximum: 16 Years
Gender: All

Health Condition(s) or Problem(s) studied

Autism

Intervention(s)

Biological: Umbilical cord mesenchymal stem cells

Primary Outcome(s)

Number of participants with adverse events [Time Frame: 89 weeks]

Secondary Outcome(s)

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]

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]

Secondary ID(s)

TBS-UCMSC-ASD001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors