- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03099239
hCT-MSCs for Children With Autism Spectrum Disorder (ASD) (hCT-MSCs)
A Phase I Study of hCT-MSC, An Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
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North Carolina
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Durham, North Carolina, United States, 27705
- Duke University Medical Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
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
- Significant hematologic abnormalities defined as: Hemoglobin <10.0 g/dL, WBC < 3,000 cells/mL, ALC <1000/uL, Platelets <150 x 10e9/uL
- Evidence of clinically relevant physical dysmorphology indicative of a genetic syndrome as assessed by the PIs or other investigators, including a medical geneticist and psychiatrists trained in identifying dysmorphic features associated with neurodevelopmental conditions.
Current/Prior Therapy:
a. History of prior cell therapy b. Current or prior use of IVIG or other anti-inflammatory medications with the exception of NSAIDs c. Current or prior immunosuppressive therapy i. No systemic steroid therapy that has lasted >2 weeks, and no systemic steroids within 3 months prior to enrollment. Topical and inhaled steroids are permitted.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Single hCT-MSC infusion
Subjects 1-3 will receive a single infusion of hCT-MSCs.
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hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.
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Experimental: Two hCT-MSC infusions
Subjects 4-6 will receive two infusions of hCT-MSCs.
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hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.
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Experimental: Three hCT-MSC infusions
Subjects 6-12 will receive three infusions of hCT-MSCs.
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hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of Infusion reactions
Time Frame: 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.
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Patients will be assessed for infusion reactions.
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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.
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Incidence of Infections
Time Frame: 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.
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Patients will be assessed for infections.
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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.
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Geraldine Dawson, PhD, Duke University
- Principal Investigator: Jessica Sun, MD, Duke University
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- Pro00079421
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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