NCT03099239

hCT-MSCs for Children With Autism Spectrum Disorder (ASD)

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Joanne Kurtzberg, MD · Academic / Other
• Sex: All
• Age: 2 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this Phase 1 study is to determine the safety of one, two, and three intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), administered every two months, in children with autism spectrum disorder (ASD).

Detailed description

This study is a phase I, prospective, open-label trial designed to assess the safety of one, two, and three intravenous doses of hCT-MSC in young children with ASD. Children ages two to 11 years with ASD will be eligible to participate. All participants will receive intravenous infusion(s) of CTCs. The first cohort of three patients will receive a single dose. If there are no safety concerns, the second cohort of three patients will receive two doses, given two months apart. The third cohort will consist of six patients, each of whom will receive three hCT-MSC infusions with a two-month interval between doses. All participants will have an initial clinical evaluation to verify the diagnosis of ASD and confirm protocol eligibility. The main endpoint is safety, for which acute infusion reactions and incidence of infections will be assessed. ASD-specific outcome measures, described below, will be assessed at baseline and six months from baseline and results will be described.

Conditions

• Autism
• Autism Spectrum Disorder
• ASD

Interventions

Type	Name	Description
BIOLOGICAL	hCT-MSC infusion	hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.

Timeline

• Start date: 2017-06-06
• Primary completion: 2019-06-10
• Completion: 2019-06-10
• First posted: 2017-04-04
• Last updated: 2019-12-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study