NCT04089579

hCT-MSC in Children With Autism Spectrum Disorder

A Phase II Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 137 (actual)
• Sponsor: Joanne Kurtzberg, MD · Academic / Other
• Sex: All
• Age: 4 Years – 11 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Autism
• Autism Spectrum Disorder

Interventions

Type	Name	Description
BIOLOGICAL	Cord Tissue Mesenchymal Stromal Cells	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.
OTHER	Placebo Infusion	Placebo comparative infusion

Timeline

• Start date: 2020-10-12
• Primary completion: 2023-05-02
• Completion: 2024-02-01
• First posted: 2019-09-13
• Last updated: 2024-02-14

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study