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

Completed · Phase 1

Conditions studied: Autism, Autism Spectrum Disorder, ASD

In brief

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).

Key facts

Study ID: NCT03099239
Run by: Joanne Kurtzberg, MD
People needed: 12
Starts: 2017-06-06
Expected to finish: 2019-06-10
Last updated by the study team: 2019-12-03

Who can join

Age: 2 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

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
Known abnormal thyroid function (patients with treated hypothyroidism with a normal TSH may be included)
Known mitochondrial dysfunction
History of unstable epilepsy or uncontrolled seizure disorder, infantile spasms, Lennox Gastaut syndrome, Dravet syndrome, or other similar chronic seizure disorder
Active malignancy or prior malignancy that was treated with chemotherapy
History of a primary immunodeficiency disorder
History of autoimmune cytopenias (i.e., ITP, AIHA)
Coexisting medical condition that would place the child at increased risk for complications of study procedures
Concurrent genetic or acquired disease or comorbidity(ies) that could require a future stem cell transplant
Significant sensory (e.g., blindness, deafness, uncorrected hearing impairment) or motor (e.g., cerebral palsy) impairment
Impaired renal or liver function as determined by serum creatinine >1.5mg/dL or total bilirubin >1.3mg/dL, except in patients with known Gilbert's disease

Where it is running

Duke University Medical Center — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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