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

NCT03099239 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: Pro00079421
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Autism; Autism Spectrum Disorder; ASD

Outcome Measures

Primary Outcomes (2)

• Incidence of Infusion reactions

  Patients will be assessed for infusion reactions.

  Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.

• Incidence of Infections

  Patients will be assessed for infections.

  Assessed for a significant change at the time of each infusion, 24 hours after each infusion, 7-10 days after each infusion, 6 and 12 months after the final infusion.

Study Arms (3)

Single hCT-MSC infusion

EXPERIMENTAL

Subjects 1-3 will receive a single infusion of hCT-MSCs.

Biological: hCT-MSC infusion

Two hCT-MSC infusions

EXPERIMENTAL

Subjects 4-6 will receive two infusions of hCT-MSCs.

Biological: hCT-MSC infusion

Three hCT-MSC infusions

EXPERIMENTAL

Subjects 6-12 will receive three infusions of hCT-MSCs.

Biological: hCT-MSC infusion

Interventions

hCT-MSC infusion BIOLOGICAL

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

Single hCT-MSC infusion; Three hCT-MSC infusions; Two hCT-MSC infusions

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age ≥ 2 years to ≤ 12 years (11 years, 364 days) at the time of consent
• Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist with a moderate severity level of ASD as reflected by SRS score ≥ 66 and CGI-S severity score of ≥ 4.
• 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
• Normal absolute lymphocyte count (≥1500/uL)
• Participant and parent/guardian are English speaking
• Able to travel to Duke University up to four times (baseline, every two months for subsequent infusions, and 6 months after initial infusion), and parent/guardian is able to participate in interim surveys and interviews
• Parental consent

You may not qualify if:

• General:
• Review of medical records indicates ASD diagnosis not likely
• Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder associated with bipolar disorder, Tourette syndrome
• Screening data suggests that participant would not be able to comply with the requirements of the study procedures as assessed by the study team
• Family is unwilling or unable to commit to participation in all study-related assessments, including protocol follow up
• Sibling is enrolled in this (Duke hCT-MSC) study
• Genetic:
• Records indicate that child 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
• Evaluation by geneticist (performed locally as standard of care or remotely by the study geneticist via review of available data - minimally medical records, photos, Fragile X and CMA testing) indicates a genetic cause for ASD.
• Infectious:
• Known active CNS infection
• Evidence of uncontrolled infection based on records or clinical assessment
• Known HIV positivity
• Medical:
• Known metabolic disorder

Sponsors & Collaborators

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

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Geraldine Dawson, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

• Jessica Sun, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Scientific Officer, Robertson Clinical and Translational Cell Therapy Program; Director, Pediatric Blood and Marrow Transplant Program

Study Record Dates

• First Submitted: March 24, 2017
• First Posted: April 4, 2017
• Study Start: June 6, 2017
• Primary Completion: June 10, 2019
• Study Completion: June 10, 2019
• Last Updated: December 3, 2019

Record last verified: 2019-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)