hCT-MSC in Children With Autism Spectrum Disorder (IMPACT)
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|ClinicalTrials.gov Identifier: NCT04089579|
Recruitment Status : Active, not recruiting
First Posted : September 13, 2019
Last Update Posted : October 28, 2022
|Condition or disease||Intervention/treatment||Phase|
|Autism Autism Spectrum Disorder||Biological: Cord Tissue Mesenchymal Stromal Cells Other: Placebo Infusion||Phase 2|
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 4-11 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. 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||164 participants|
|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|
|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 :||October 12, 2020|
|Estimated Primary Completion Date :||April 1, 2023|
|Estimated Study Completion Date :||October 1, 2023|
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
Other: Placebo Infusion
Placebo comparative infusion
- 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.
- Change in VABS-3 Socialization Standard Score [ Time Frame: Baseline, 6 months ]Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score
- Change in VABS-3 Communication Standard Score [ Time Frame: Baseline, 6 months ]Change in VABS-3 (Vineland Adaptive Behavior Scales) Communication Standard Score
- Change in CGI-Severity score [ Time Frame: Baseline, 6 months ]Clinical Global Impression- Severity Scale
- CGI-Intervention score [ Time Frame: Baseline, 6 months ]Clinical Global Impression- Impression
- Change in the Pediatric Quality of Life Scale [ Time Frame: Baseline, 6 months ]Pediatric Quality of Life Scale, raw scale range of 0-2300 with higher scores indicating a higher quality of life (better outcome)
- Incidence and severity of infusion reactions [ Time Frame: Baseline, 6 months ]Assess for infusion reactions
- Incidence and severity of product-related infections [ Time Frame: Baseline, 6 months ]Assess for infections directly related to the study product infusions
- Evidence of formation of anti-HLA antibodies [ Time Frame: Baseline, 6 months, 12 months ]Assess for anti-HLA antibodies
- Incidence and severity of graft versus host disease [ Time Frame: 6 months, 12 months ]Assess for signs and symptoms of graft versus host disease
- 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
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
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27705|
|Principal Investigator:||Joanne Kurtzberg, MD||Duke University|
|Principal Investigator:||Geraldine Dawson, PhD||Duke University|
|Principal Investigator:||Jessica Sun, MD||Duke University|