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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03099239
First Posted: April 4, 2017
Last Update Posted: October 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
The Marcus Foundation
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University Medical Center
  Purpose
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).

Condition Intervention Phase
Autism Autism Spectrum Disorder ASD Biological: hCT-MSC infusion Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study of hCT-MSC, An Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder

Resource links provided by NLM:


Further study details as provided by Joanne Kurtzberg, MD, Duke University Medical Center:

Primary Outcome Measures:
  • 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. ]
    Patients will be assessed for infusion reactions.

  • Incidence of Infections [ Time Frame: Assessed for a signifiacnt 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. ]
    Patients will be assessed for infections.


Enrollment: 12
Actual Study Start Date: June 6, 2017
Estimated Study Completion Date: January 3, 2019
Estimated Primary Completion Date: January 3, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single hCT-MSC infusion
Subjects 1-3 will receive a single infusion of hCT-MSCs.
Biological: hCT-MSC infusion
hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.
Experimental: Two hCT-MSC infusions
Subjects 4-6 will receive two infusions of hCT-MSCs.
Biological: hCT-MSC infusion
hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.
Experimental: Three hCT-MSC infusions
Subjects 6-12 will receive three infusions of hCT-MSCs.
Biological: hCT-MSC infusion
hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked.

Detailed Description:
This study is a phase I, prospective, open-label trial designed to assess the safety of one, two, and three intravenous doses of hCT-MSC in young children with ASD. Children ages two to 11 years with ASD will be eligible to participate. All participants will receive intravenous infusion(s) of CTCs. The first cohort of three patients will receive a single dose. If there are no safety concerns, the second cohort of three patients will receive two doses, given two months apart. The third cohort will consist of six patients, each of whom will receive three hCT-MSC infusions with a two-month interval between doses. All participants will have an initial clinical evaluation to verify the diagnosis of ASD and confirm protocol eligibility. The main endpoint is safety, for which acute infusion reactions and incidence of infections will be assessed. ASD-specific outcome measures, described below, will be assessed at baseline and six months from baseline and results will be described.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age ≥ 2 years to ≤ 12 years (11 years, 364 days) at the time of consent
  2. 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.
  3. Fragile X testing performed and negative; CMA and/or whole exome sequencing performed and results not linked to autism diagnosis
  4. Stable on current psychiatric medication regimen (dose and dosing schedule) for at least 2 months prior to infusion of study product
  5. Normal absolute lymphocyte count (≥1500/uL)
  6. Participant and parent/guardian are English speaking
  7. 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
  8. Parental consent

Exclusion Criteria:

  1. General:

    1. Review of medical records indicates ASD diagnosis not likely
    2. Known diagnosis of any of the following coexisting psychiatric conditions: depression, bipolar disorder, schizophrenia, obsessive compulsive disorder associated with bipolar disorder, Tourette syndrome
    3. Screening data suggests that participant would not be able to comply with the requirements of the study procedures as assessed by the study team
    4. Family is unwilling or unable to commit to participation in all study-related assessments, including protocol follow up
    5. Sibling is enrolled in this (Duke hCT-MSC) study
  2. Genetic:

    1. 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
    2. 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.
  3. Infectious:

    1. Known active CNS infection
    2. Evidence of uncontrolled infection based on records or clinical assessment
    3. Known HIV positivity
  4. Medical:

    1. Known metabolic disorder
    2. Known abnormal thyroid function (patients with treated hypothyroidism with a normal TSH may be included)
    3. Known mitochondrial dysfunction
    4. History of unstable epilepsy or uncontrolled seizure disorder, infantile spasms, Lennox Gastaut syndrome, Dravet syndrome, or other similar chronic seizure disorder
    5. Active malignancy or prior malignancy that was treated with chemotherapy
    6. History of a primary immunodeficiency disorder
    7. History of autoimmune cytopenias (i.e., ITP, AIHA)
    8. Coexisting medical condition that would place the child at increased risk for complications of study procedures
    9. Concurrent genetic or acquired disease or comorbidity(ies) that could require a future stem cell transplant
    10. Significant sensory (e.g., blindness, deafness, uncorrected hearing impairment) or motor (e.g., cerebral palsy) impairment
    11. 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
    12. Significant hematologic abnormalities defined as: Hemoglobin <10.0 g/dL, WBC < 3,000 cells/mL, ALC <1000/uL, Platelets <150 x 10e9/uL
    13. 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 dysmporphic features associated with neurodevelopmental conditions.
  5. 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.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03099239


Locations
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Joanne Kurtzberg, MD
The Marcus Foundation
Investigators
Principal Investigator: Joanne Kurtzberg, MD Duke University
Principal Investigator: Geraldine Dawson, PhD Duke University
Principal Investigator: Jessica Sun, MD Duke University
  More Information

Responsible Party: Joanne Kurtzberg, MD, Chief Scientific Officer, Robertson Clinical and Translational Cell Therapy Program; Director, Pediatric Blood and Marrow Transplant Program, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT03099239     History of Changes
Other Study ID Numbers: Pro00079421
First Submitted: March 24, 2017
First Posted: April 4, 2017
Last Update Posted: October 23, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders