Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)

NCT02176317 | Completed Phase 1 DMC

• 1 other identifier: Pro00052449
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism Spectrum Disorder

Keywords

Autism Spectrum Disorder; ASD; Umbilical cord blood; Autologous cord blood; cord blood

Outcome Measures

Primary Outcomes (1)

• Number of participants with non-serious and serious adverse events.

  up to 12 months

Secondary Outcomes (1)

• The primary efficacy measure will be change in the Vineland Adaptive Behavior Scale- II

  up to 12 months

Other Outcomes (14)

• Change in Pervasive Developmental Disorder- Behavior Inventory

  up to 12 months

• Change in Repetitive Behavior Scal

  up to 12 months

• Change in Sensory Experience Questionnaire

  up to 12 months

Study Arms (1)

Autologous Umbilical Cord Blood (UCB)

EXPERIMENTAL

All participants will receive a single intravenous (into the vein) infusion of autologous umbilical cord blood cells.

Biological: Autologous Umbilical Cord Blood

Interventions

Autologous Umbilical Cord Blood BIOLOGICAL

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

Autologous Umbilical Cord Blood (UCB)

Eligibility Criteria

• Age: 24 Months - 72 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Duke University: lead
• The Marcus Foundation: collaborator
• PerkinElmer, Inc.: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Related Publications (1)

• 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.

  PMID: 32616759 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: June 16, 2014
• First Posted: June 27, 2014
• Study Start: June 1, 2014
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: December 3, 2019

Record last verified: 2019-12

Locations

US (1)