hCT-MSC in Children With Autism Spectrum Disorder

Phase 2 Completed 137 enrolled Joanne Kurtzberg, MD Cryo-Cell International · collab The Marcus Foundation · collab

RandomizedParallel-groupQuadruple-blindTreatment

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).

Timeline

Start: 2020-10-12
Primary completion: 2023-05-02
Completion: 2024-02-01

Outcome

Missed primary endpoint

registry MSC (Mesenchymal Stromal Cells) Infusion vs Placebo Infusion; p = 0.35; Mean Difference (Net) 1.32 (95% CI -0.88 to 3.51) NCT04089579 ↗

Publications

Background Chatham CH, Taylor KI, Charman T, Liogier D'ardhuy X, Eule E, Fedele A, Hardan AY, Loth E, Murtagh L, Del Valle Rubido M, San Jose Caceres A, Sevigny J, Sikich L, Snyder L, Tillmann JE, Ventola PE, Walton-Bowen KL, Wang PP, Willgoss T, Bolognani F. Adaptive behavior in autism: Minimal clinically important differences on the Vineland-II. Autism Res. 2018 Feb;11(2):270-283. doi: 10.1002/aur.1874. Epub 2017 Sep 21.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cord Tissue Mesenchymal Stromal Cells	Cell therapy	6e+06 cells/kg	Intravenous