hCT-MSC in Children With Autism Spectrum Disorder

Phase 2

Completed

• Conditions: Autism Spectrum Disorder; Autism
• Interventions: Biological: Cord Tissue Mesenchymal Stromal Cells; Other: Placebo Infusion

• Registration Number: NCT04089579
• Lead Sponsor: Joanne Kurtzberg, MD

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).

Detailed Description

The purpose of this double blinded Phase II study is to determine the efficacy of human umbilical cord tissue-derived mesencymal stromal cells (hCT-MSC), administered in two different dosing strategies, in children with autism spectrum disorder (ASD).

This study will be enrolling children with ASD, aging 4-11 years of age. Qualifying subjects will undergo neuropsychological evaluation, EEG testing, eye tracking, CVA assessments, and infusion of study product. Subjects will be randomized to one of two study arms; 1) a single infusion of 6.0x106 cells/Kg at baseline, followed by a blinded placebo infusion at six months or, 2) Placebo infusion at baseline, followed by an intravenous dose of 6x106 cells/Kg at six months.

The primary endpoint of this study is the change in social communication skill from baseline to six months. The potential risks associated with infusion of MSCs include a reaction to the product (rash, shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth, throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization.

Study Timeline

• Study First Submitted: 2019-09-12
• Study First Submitted QC: 2019-09-12
• Study First Posted: 2019-09-13
• Study Start: 2020-10-12
• Primary Completion: 2023-05-02
• Status Verified: 2024-02
• Study Completion: 2024-02-01
• Last Update Submitted: 2024-02-13
• Last Update Posted: 2024-02-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 137

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
MSC	Cord Tissue Mesenchymal Stromal Cells	One dose of 6x10e6 cells/kg administered intravenously.
Placebo Infusion	Placebo Infusion	Placebo infusion

Primary Outcome Measures

Name	Time	Method
Change on the Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales	Baseline, 6 months	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.

Secondary Outcome Measures

Name	Time	Method
Change in CGI-Severity score	Baseline, 6 months	Clinical Global Impression- Severity Scale
Change in VABS-3 Socialization Standard Score	Baseline, 6 months	Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score
Change in the Pediatric Quality of Life Scale	Baseline, 6 months	Pediatric Quality of Life Scale, raw scale range of 0-2300 with higher scores indicating a higher quality of life (better outcome)
Change in VABS-3 Communication Standard Score	Baseline, 6 months	Change in VABS-3 (Vineland Adaptive Behavior Scales) Communication Standard Score
CGI-Intervention score	Baseline, 6 months	Clinical Global Impression- Impression

Trial Locations

Locations (1)

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States