Cord Blood Infusion for Children With Autism Spectrum Disorder

NCT02847182 | Completed Phase 2 Results DMC

• 1 other identifier: Pro00070514
• Study Type: interventional
• Target: 180
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single site, prospective, randomized, double-blind study of a single intravenous autologous or allogeneic, unrelated cord blood (CB) infusion in children ages 2-7 years with Autism Spectrum Disorder (ASD). Participants will be randomly assigned to Sequence A, consisting of a single infusion of CB cells at baseline followed 6 months later by a single infusion of placebo, or Sequence B, consisting of an infusion of placebo at baseline followed 6 months later by an infusion of CB cells. All participants will ultimately be treated with CB cells at some point during the study. Participants with an available qualified autologous CB unit will receive autologous cells, and those without a suitable autologous CB unit available will receive cells from a ≥4/6 HLA-matched, ABO-matched allogeneic, unrelated donor CB unit from the Carolinas Cord Blood Bank. All infusions will be double-blinded. The primary outcomes will be assessed 6 months after the initial infusion in the sequence. Additional testing for secondary exploratory analyses will be performed at 12 months. Duration of study participation will be 12 months from the time of baseline infusion.

Conditions

Autism Spectrum Disorder; ASD; Autism; PDD

Outcome Measures

Primary Outcomes (1)

• Change in Social Communication as Measured by the Vineland Adaptive Behavior Scales, Third Edition (VABS-3)

  The Vineland Adaptive Behavior Scales, Third Edition (VABS-3) Socialization domain standard score has mean=100 and standard deviation=15 (range: 20-140). Higher scores indicate better developed adaptive social behavior. The change in the Socialization domain standard score was calculated for each participant from Baseline to Month 6. Changes in the Socialization standard score are indicative of skill acquisition relative to chronologically aged peers of the same sex. Thus, a zero (no change) represents change consistent with what is expected. An increase represents acquisition of more skills over time than would be expected. Participants who experience a decrease in Socialization standard score may still have acquired skills although not at the rate expected based on their age and sex.

  Baseline, 6 months

Secondary Outcomes (32)

• Change in Vineland Socialization Domain Raw Score

  Baseline, 6 months

• Change in Vineland Socialization Domain Age Equivalent

  Baseline, 6 months

• Change in Pervasive Developmental Disorder Behavior Inventory (PDD-BI) Composite Standard Score (Parent Questionnaire)

  Baseline, 6 months

• Change in Clinical Global Impressions - Severity of Illness (CGI-S) Score, Clinician Assessment

  Baseline, 6 months

• Clinical Global Impressions - Global Improvement (CGI-I) Score, Clinician Assessment

  Baseline, 6 months

Study Arms (2)

Cord Blood Infusion (best source)

EXPERIMENTAL

Subjects will be randomized to receive a cord blood infusion at the baseline or 6 month visit. The cord blood will be autologous (if available) or unrelated cord blood.

Biological: Cord Blood Infusion; Biological: Placebo

Placebo Infusion

PLACEBO COMPARATOR

Subjects will be randomized to receive a placebo infusion at the baseline or 6 month visit. The placebo is an acellular media product similar in both appearance and odor.

Biological: Cord Blood Infusion; Biological: Placebo

Interventions

Cord Blood Infusion BIOLOGICAL

Cord Blood Infusion (best source); Placebo Infusion

Placebo BIOLOGICAL

Cord Blood Infusion (best source); Placebo Infusion

Eligibility Criteria

• Age: 2 Years - 7 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age ≥ 2 years to ≤ 7 years (7 years, 364 days) at the time of visit 1
• Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist
• Fragile X testing performed and negative
• Available and qualified umbilical cord blood unit with a minimum banked total nucleated cell dose of ≥ 2.5 x 107 cells/kg that meets criteria outlined in Section 6.0, either:
• Autologous umbilical cord blood unit OR
• ≥4/6 HLA-matched and ABO/Rh-matched allogeneic unrelated umbilical cord blood unit from the Carolinas Cord Blood Bank
• Stable on current psychiatric medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
• Normal absolute lymphocyte count (≥1500/uL)
• Participant and parent/guardian are English speaking
• Able to travel to Duke University two times (baseline and 6 months post-baseline), and parent/guardian is able to participate in interim surveys and interviews
• Parental consent

You may not qualify if:

• General:
• Review of medical records indicates ASD diagnosis not likely
• Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder, Tourette syndrome
• Screening data suggests that participant would not be able to comply with the requirements of the study procedures, including study outcome measures, as assessed by the study team
• Family is unwilling or unable to commit to participation in all study-related assessments, including follow up for approximately 12 months
• Sibling is enrolled in this (DukeACT) study
• Genetic:
• Records indicate that child has a known genetic syndrome such as (but not limited to) Fragile X syndrome, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cystic fibrosis, muscular dystrophy b. Known pathogenic copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)
• Infectious:
• Known active central nervous system infection
• Evidence of uncontrolled infection based on records or clinical assessment
• HIV positivity
• Medical:
• Known metabolic disorder
• Known mitochondrial dysfunction

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Related Publications (1)

• Simhal AK, Carpenter KLH, Kurtzberg J, Song A, Tannenbaum A, Zhang L, Sapiro G, Dawson G. Changes in the geometry and robustness of diffusion tensor imaging networks: Secondary analysis from a randomized controlled trial of young autistic children receiving an umbilical cord blood infusion. Front Psychiatry. 2022 Oct 20;13:1026279. doi: 10.3389/fpsyt.2022.1026279. eCollection 2022.

  PMID: 36353577 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Jesse D. Troy, PhD, MPH
• Organization: Duke University

cordbloodtherapyinfo@dm.duke.edu

919-668-1102

Study Officials

• Joanne Kurtzberg, MD

  Duke Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Scientific Officer, Robertson Clinical and Translational Cell Therapy Program; Director, Pediatric Blood and Marrow Transplant Program and Carolinas Cord Blood Bank

Study Record Dates

• First Submitted: July 25, 2016
• First Posted: July 28, 2016
• Study Start: September 1, 2016
• Primary Completion: August 19, 2018
• Study Completion: May 1, 2019
• Last Updated: June 9, 2020
• Results First Posted: December 26, 2019

Record last verified: 2020-05

Locations

US (1)