hCT-MSC Infusion in Adults With Autism Spectrum Disorder

NCT04484077 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PRO00104691
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Autism Spectrum Disorder; Autism

Keywords

autism; MSC; hCT-MSC; mesenchymal stromal cells

Outcome Measures

Primary Outcomes (5)

• Incidence of infusion reactions

  cumulative incidence as measured by clinical examination and patient interview

  Baseline through 10 days post infusion

• Incidence of product-related infections

  cumulative incidence as measured by patient interview and questionnaire

  Baseline through 12 months

• Evidence of formation of anti-HLA antibodies

  change from baseline to 6 and 12 months post infusion as measured by PRA testing

  Baseline, 6 months, 12 months

• Incidence of graft vs. host disease

  cumulative incidence as measured by patient interview and questionnaire

  Baseline through 12 months

• Incidence of unexpected adverse events, by severity and relation to study

  cumulative incidence as measured by patient questionnaire and clinical labs

  Baseline through 12 months

Secondary Outcomes (1)

• The Vineland Adaptive Behavior Scale Interview, 3rd Edition, Comprehensive interview form

  Baseline and 6 months

Study Arms (1)

hCT-MSC infusion

EXPERIMENTAL

A single, intravenous infusion of hCT-MSCs. Targeted dose is 2x10\^6 cells/kg with a maximum dose of 10 x 10\^7 cells/kg.

Biological: hCT-MSC

Interventions

hCT-MSC BIOLOGICAL

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

hCT-MSC infusion

Eligibility Criteria

• Age: 18 Years - 34 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age ≥18 to \< 35 years (34 years, 364 days) at the time of consent
• Confirmed pre-existing diagnosis of ASD in the individual's educational and/or medical record
• Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
• Stable on current psychiatric medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product, with no intention of changing or beginning new psychiatric medications or behavioral treatments during the duration of the study.
• Normal absolute lymphocyte count (≥1200/uL for African American participants and ≥1500/uL for all other participants)
• General Ability Index of ≥70, confirmed through cognitive testing completed by study personnel to establish eligibility
• Participant and Parent are English speaking
• Able to travel to Duke University for baseline visit.
• Participant has a parent who spends four or more hours a week with the participant, who is able and willing to participate in study visits and interim surveys and interviews
• Informed consent of the participant and parent (participants must have legal authority regarding their own medical care and a parent or legally authorized representative may not consent on their behalf)

You may not qualify if:

• General:
• Review of medical records indicates ASD diagnosis and IQ \> 70 unlikely.
• Known or suspected diagnosis of any of the following coexisting psychiatric conditions: bipolar disorder, schizophrenia, Tourette syndrome, and/or any co-occurring psychiatric disorder that the investigator believes would interfere with accurate completion of study instruments and/or current (within the past year) evidence of suicidality as assessed by an interview with participant and/or parent which includes the Columbia Suicide Severity Rating Scale, and a review of responses on the Behavioral and Symptom Identification Scale (BASIS-24).
• Screening data or in-person evaluations suggest that participant would not be able to comply with the requirements of the study procedures as assessed by the study team
• Parent and/or participant is unwilling or unable to commit to participation in all study-related assessments, including protocol follow up
• Sibling is enrolled in this (Duke AIMs) study
• Genetic:
• Records indicate that the participant has a known genetic syndrome such as (but not limited to) Fragile X syndrome, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cystic fibrosis, muscular dystrophy or a genetic defect definitively known to be associated with ASD
• Known pathogenic mutation or copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)
• Infectious:
• Known active CNS infection
• Evidence of uncontrolled infection based on records or clinical assessment
• Known HIV positivity
• Exposure to COVID-19 in the preceding 14 days or positive COVID-19 test in the previous 28 days. Subjects with a past history of infection with COVID-19 must be symptom-free for 14 days prior to the initial visit.
• Medical:

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead
• The Marcus Foundation: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Jessica Sun, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Jerome Harris Distinguished Professor of Pediatrics; Professor of Pathology; Director, Marcus Center for Cellular Cures; Director, Pediatric Blood and Marrow Transplant Program; Director, Carolinas Cord Blood Bank

Study Record Dates

• First Submitted: July 16, 2020
• First Posted: July 23, 2020
• Study Start: January 24, 2022
• Primary Completion: April 29, 2024
• Study Completion: April 29, 2024
• Last Updated: July 23, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)