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Trial record 14 of 19 for:    stem cells and autism

hCT-MSC in Children With Autism Spectrum Disorder (IMPACT)

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ClinicalTrials.gov Identifier: NCT04089579
Recruitment Status : Recruiting
First Posted : September 13, 2019
Last Update Posted : November 14, 2019
Sponsor:
Collaborator:
The Marcus Foundation
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Brief Summary:
The purpose of this Phase II study is to determine the efficacy of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) for improving social communication abilities in children with autism spectrum disorder (ASD).

Condition or disease Intervention/treatment Phase
Autism Autism Spectrum Disorder Biological: Cord Tissue Mesenchymal Stromal Cells Other: Placebo Infusion Phase 2

Detailed Description:

The purpose of this double blinded Phase II study is to determine the efficacy of human umbilical cord tissue-derived mesencymal stromal cells (hCT-MSC), administered in two different dosing strategies, in children with autism spectrum disorder (ASD).

This study will be enrolling children with ASD, aging 4-8 years of age. Qualifying subjects will undergo neuropsychological evaluation, EEG testing, eye tracking, CVA assessments, and infusion of study product. Subjects will be randomized to one of two study arms; 1) a single infusion of 6.0x106 cells/Kg at baseline, followed by a blinded placebo infusion at six months or, 2) Placebo infusion at baseline, followed by an intravenous dose of 6x106 cells/Kg at six months.

The primary endpoint of this study is the change in social communication skill from baseline to six months, as measured by the Vineland Adaptive Behavior Scale-Thirdy Edition (VABS-3). The potential risks associated with infusion of MSCs include a reaction to the product (rash, shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth, throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 164 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: This study is a phase II, prospective, randomized, blinded, cross over, clinical trial designed to assess the efficacy of intravenous dosing of hCT-MSC for improving social communication abilities in young children with ASD. All participants will ultimately be treated with hCT-MSC. Participants randomized to arm A will each receive a single intravenous dose of 6x106 hCT-MSC per kilogram at baseline, followed by a placebo infusion at six months. Participants randomized to arm B will each receive a placebo infusion at baseline, followed by an intravenous dose of 6x106 hCT-MSC per kilogram at six months.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Blinded infusion
Primary Purpose: Treatment
Official Title: A Phase II Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder
Actual Study Start Date : November 4, 2019
Estimated Primary Completion Date : November 1, 2021
Estimated Study Completion Date : April 1, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: MSC
One dose of 6x10e6 cells/kg administered intravenously.
Biological: Cord Tissue Mesenchymal Stromal Cells
Human Umbilical Cord Tissue Derived Mesenchymal Stromal Cells (hCT-MSC), isolated and expanded from umbilical cord tissue from allogeneic unrelated donors. One dose of 6x10e6 cells/kg administered intravenously.

Placebo Comparator: Placebo Infusion
Placebo infusion
Other: Placebo Infusion
Placebo comparative infusion




Primary Outcome Measures :
  1. Change on the Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales [ Time Frame: Baseline, 6 months ]
    The primary outcome measure is the mean of the change on the Socialization and Communication Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3). The primary endpoint is the change on this outcome measure from baseline to six months.


Secondary Outcome Measures :
  1. Change in VABS-3 Socialization Standard Score [ Time Frame: Baseline, 6 months ]
    Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score

  2. Change in VABS-3 Communication Standard Score [ Time Frame: Baseline, 6 months ]
    Change in VABS-3 (Vineland Adaptive Behavior Scales) Communication Standard Score

  3. Change in CGI-Severity score [ Time Frame: Baseline, 6 months ]
    Clinical Global Impression- Severity Scale

  4. CGI-Intervention score [ Time Frame: Baseline, 6 months ]
    Clinical Global Impression- Impression


Other Outcome Measures:
  1. Incidence and severity of infusion reactions [ Time Frame: Baseline, 6 months ]
    Assess for infusion reactions

  2. Incidence and severity of product-related infections [ Time Frame: Baseline, 6 months ]
    Assess for infections directly related to the study product infusions

  3. Evidence of formation of anti-HLA antibodies [ Time Frame: Baseline, 6 months, 12 months ]
    Assess for anti-HLA antibodies

  4. Incidence and severity of graft versus host disease [ Time Frame: 6 months, 12 months ]
    Assess for signs and symptoms of graft versus host disease

  5. Incidence and severity of unexpected adverse events related to the study product [ Time Frame: Baseline, 6 months, 12 months ]
    Assess for study related and unexpected adverse events



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age ≥ 4 years to ≤ 8 years (7 years, 364 days) at the time of consent
  2. Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using the DSM-5 Checklist as informed by the Autism Diagnostic Observation Scale - 2 (ADOS-2) and Autism Diagnostic Interview-Revised (ADI-R).
  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 (≥1200/uL for African American participants and ≥1500/uL for all other participants)
  6. Full Scale IQ ≥70 per local records and confirmed through cognitive testing completed by study personnel
  7. Participant and parent/guardian are English speaking
  8. Able to travel to Duke University two times (baseline, six months), and parent/guardian is able to participate in interim surveys and interviews
  9. Parental/guardian consent

Exclusion Criteria:

  1. General:

    1. Review of medical records indicates ASD diagnosis and/or IQ > 70 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. Known pathogenic mutation or copy number variation (CNV) associated with ASD (e.g., 16p11.2, 15q13.2, 2q13.3)
  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 mitochondrial dysfunction
    3. History of unstable epilepsy or uncontrolled seizure disorder, infantile spasms, Lennox Gastaut syndrome, Dravet syndrome, or other similar chronic seizure disorder
    4. Active malignancy or prior malignancy that was treated with chemotherapy
    5. History of a primary immunodeficiency disorder
    6. History of autoimmune cytopenias (i.e., ITP, AIHA)
    7. Coexisting medical condition that would place the child at increased risk for complications of study procedures
    8. Concurrent genetic or acquired disease or comorbidity(ies) that could require a future stem cell transplant
    9. Significant sensory (e.g., blindness, deafness, uncorrected hearing impairment) or motor (e.g., cerebral palsy) impairment
    10. 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
    11. Significant hematologic abnormalities defined as: Hemoglobin <10.0 g/dL, Platelets <150 x 10e9/uL, WBC <3,000 cells/mL, ALC <1200/uL for African Americans or <1500/uL for all other participants.
    12. 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.
  5. Current/Prior Therapy:

    a. Availability of a banked, qualified autologous cord blood unit or parents deferred use of qualified, autologous cord blood unit b. History of prior cell therapy c. Current or prior use of IVIG or other anti-inflammatory medications with the exception of NSAIDs d. 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.

    *If remote cognitive testing reveals an IQ > 70 but testing at the baseline visit reveals an IQ >65, the patient will be deemed eligible. This is due to the margin of error that is present with this type of testing.


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): NCT04089579


Contacts
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Contact: Nick Chapman 9196681102 cordbloodtherapyinfo@dm.duke.edu
Contact: Kerry Hoyle 9196681102 cordbloodtherapyinfo@dm.duke.edu

Locations
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United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27705
Contact: Jessica Sun, MD       cordbloodtherapyinfo@dm.duke.edu   
Principal Investigator: Joanne Kurtzberg, MD         
Sub-Investigator: Geraldine Dawson, PhD         
Sub-Investigator: Jessica Sun, MD         
Sponsors and Collaborators
Joanne Kurtzberg, MD
The Marcus Foundation
Investigators
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Principal Investigator: Joanne Kurtzberg, MD Duke University
Principal Investigator: Geraldine Dawson, PhD Duke University
Principal Investigator: Jessica Sun, MD Duke University

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Responsible Party: Joanne Kurtzberg, MD, Professor of pediatrics, Duke University
ClinicalTrials.gov Identifier: NCT04089579     History of Changes
Other Study ID Numbers: Pro00102894
First Posted: September 13, 2019    Key Record Dates
Last Update Posted: November 14, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Joanne Kurtzberg, MD, Duke University:
Autism
Autism Spectrum Disorder
Stem cell
Mesenchymal Stromal Cells
Additional relevant MeSH terms:
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Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders