hCT-MSC in Children With Autism Spectrum Disorder

NCT04089579 | Completed Phase 2 DMC FDA Drug

• 2 other identifiers: Pro00113011Pro00102894
• Study Type: interventional
• Target: 137
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this Phase II study is to determine the efficacy of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) for improving social communication abilities in children with autism spectrum disorder (ASD).

Conditions

Autism; Autism Spectrum Disorder

Keywords

Autism; Autism Spectrum Disorder; Stem cell; Mesenchymal Stromal Cells

Outcome Measures

Primary Outcomes (1)

• Change on the Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales

  The primary outcome measure is the mean of the change on the Socialization and Communication Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3). The primary endpoint is the change on this outcome measure from baseline to six months.

  Baseline, 6 months

Secondary Outcomes (5)

• Change in VABS-3 Socialization Standard Score

  Baseline, 6 months

• Change in VABS-3 Communication Standard Score

  Baseline, 6 months

• Change in CGI-Severity score

  Baseline, 6 months

• CGI-Intervention score

  Baseline, 6 months

• Change in the Pediatric Quality of Life Scale

  Baseline, 6 months

Other Outcomes (5)

• Incidence and severity of infusion reactions

  Baseline, 6 months

• Incidence and severity of product-related infections

  Baseline, 6 months

• Evidence of formation of anti-HLA antibodies

  Baseline, 6 months, 12 months

Study Arms (2)

MSC

EXPERIMENTAL

One dose of 6x10e6 cells/kg administered intravenously.

Biological: Cord Tissue Mesenchymal Stromal Cells

Placebo Infusion

PLACEBO COMPARATOR

Placebo infusion

Other: Placebo Infusion

Interventions

Cord Tissue Mesenchymal Stromal Cells BIOLOGICAL

Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC), isolated and expanded from umbilical cord tissue from allogeneic unrelated donors. One dose of 6x10e6 cells/kg administered intravenously.

MSC

Placebo Infusion OTHER

Placebo comparative infusion

Placebo Infusion

Eligibility Criteria

• Age: 4 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age ≥ 4 years to \< 12 years (11 years, 364 days) at the time of consent
• Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist as informed by the Brief Observation of Symptoms of Autism (BOSA) and the Autism Diagnostic Interview-Revised (ADI-R)
• Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
• 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 (≥1200/uL for African American participants and ≥1500/uL for all other participants)
• GAI ≥ 65 via cognitive testing by study personnel
• Participant and parent/guardian are English speaking
• Able to travel to Duke University two times (baseline, six months), and parent/guardian is able to participate in interim surveys and interviews
• Parental/guardian consent from at least one parent/guardian

You may not qualify if:

• General:
• Review of medical records and/or screening assessments indicates ASD diagnosis and/or GAI \> 65 not confident
• Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder associated with bipolar disorder, Tourette syndrome
• Screening data suggests that participant would not be able to comply with the requirements of the study procedures as assessed by the study team
• Family is unwilling or unable to commit to participation in all study-related assessments, including protocol follow up
• Sibling is enrolled in this (Duke IMPACT) 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 or a genetic defect definitively known to be associated with ASD
• Known pathogenic mutation or copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)
• Infectious:
• Known active CNS infection
• Evidence of uncontrolled infection based on records or clinical assessment
• Known HIV positivity
• Exposure to COVID-19 in the preceding 14 days or positive COVID-19 test in the previous 28 days. Subjects with a past history of infection with COVID-19 must be symptom-free for 14 days prior to the initial visit.
• Medical:

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead
• The Marcus Foundation: collaborator
• Cryo-Cell International: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Beth Shaz, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Lauren Franz, MBChB

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Blinded infusion
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of pediatrics

Model Details: This study is a phase II, prospective, randomized, blinded, cross over, clinical trial designed to assess the efficacy of intravenous dosing of hCT-MSC for improving social communication abilities in young children with ASD. All participants will ultimately be treated with hCT-MSC. Participants randomized to arm A will each receive a single intravenous dose of 6x106 hCT-MSC per kilogram at baseline, followed by a placebo infusion at six months. Participants randomized to arm B will each receive a placebo infusion at baseline, followed by an intravenous dose of 6x106 hCT-MSC per kilogram at six months.

Study Record Dates

• First Submitted: September 12, 2019
• First Posted: September 13, 2019
• Study Start: October 12, 2020
• Primary Completion: May 2, 2023
• Study Completion: February 1, 2024
• Last Updated: February 14, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)