NCT04484077

hCT-MSC Infusion in Adults With Autism Spectrum Disorder

A PHASE I STUDY OF hCT-MSC, AN UMBILICAL CORD-DERIVED MESENCHYMAL STROMAL CELL PRODUCT, IN ADULTS WITH AUTISM SPECTRUM DISORDER

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Joanne Kurtzberg, MD · Academic / Other
• Sex: All
• Age: 18 Years – 34 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to determine the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSC is a cell product isolated from umbilical cord tissue. The cells from the cord tissue are processed and expanded in the laboratory and then infused intravenously in a single dose per participant. Participants will be ages 18-35 years, with ASD and a full-scale IQ \>70 without an identified genetic cause of autism. Participants will have an in-person baseline visit and remote follow up visits at 6 and 12 months. In addition to the primary endpoints evaluating safety, the study will evaluate changes in social communications skills after hCT-MSC administration.

Detailed description

This is a prospective, open label, phase one study to determine the safety and tolerability of a single intravenous dose of Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC) in adults with autism spectrum disorder (ASD). hCT-MSCs are manufactured from umbilical cord tissue donated by healthy mothers delivering full term babies via Cesarean Section. The cells are extracted from the cord tissue, expanded and cryopreserved (frozen). One dose of 2x10\^6 cells/kg (maximum of 10 x 10\^7) will be administered intravenously to each participant in this study. It is hypothesized that immune dysregulation and/or abnormal neuronal connectivity that adversely affects normal brain development may cause core symptomatology observed in individuals with ASD. Mesenchymal stromal cells (MSC) have demonstrated a multitude of immunomodulatory effects which are thought to be carried out via paracrine and trophic signaling. While MSCs modulate the immune response, MSCs themselves have low immunogenicity (the body does not have a strong immune reaction against them) and they do not permanently engraft in the recipient. Adults ages 18 to less than 35 years with ASD will be eligible to participate. All participants will have a screening visit that includes clinical evaluations to verify cognitive abilities and confirmation of eligibility. One dose of 2x10\^6 cells/kg (maximum of 10 x 10\^7) will be administered intravenously to each participant at a baseline visit. Participants will be admitted to the infusion center on the day of their baseline visit and vital signs (heart rate, blood pressure, temperature, respiratory rate) will be measured. Participants may request anti-anxiety medication prior to IV placement if desired. A peripheral IV will be placed and prior to the infusion, premedications (Benadryl, Solumedrol, 0.5mg/kg each) will be administered. The hCT-MSCs product will be administered intravenously over 30-60 minutes. Pulse oximetry will be monitored continuously throughout the infusion and IV fluid maintenance will be given. Participants will be discharged after 1 hour, providing all vital signs are at their baseline and they are asymptomatic with no evidence of toxicity. Participants will be evaluated the day after the infusion to assess for any infusion-related adverse reactions or complications. A phone call or email will be done to assess safety of the infusion 7-10 days after the infusion. Remote follow up will be conducted 6 months and one year after infusion. The Medical and Behavioral History Questionnaire assessing adverse events will be obtained at baseline, 6 and 12-months. The main endpoint is safety, and acute infusion reactions, incidence of infections, and markers of alloimmunization will be assessed. Social communication will be assessed at baseline and 6 months post product administration.

Conditions

• Autism Spectrum Disorder
• Autism

Interventions

Type	Name	Description
BIOLOGICAL	hCT-MSC	2x10\^6 hCT-MSC/kg suspended in plasmalyte-A, 5% HSA, and residual DMSO/dextran administered intravenously over 30-60 minutes via syringe pump

Timeline

• Start date: 2022-01-24
• Primary completion: 2024-04-29
• Completion: 2024-04-29
• First posted: 2020-07-23
• Last updated: 2024-07-23

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study