Trial Outcomes & Findings for Open Label Phase I hCT-MSC in Toddlers With Autism Spectrum Disorder (NCT NCT04294290)

NCT ID: NCT04294290

Last Updated: 2025-02-24

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 12 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2025-02-24

Participant Flow

Participant milestones

Measure	hCT-MSC Infusion hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Open Label Phase I hCT-MSC in Toddlers With Autism Spectrum Disorder

Baseline characteristics by cohort

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Age, Continuous	39.3 months STANDARD_DEVIATION 7.1 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	4 Participants n=5 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	7 Participants n=5 Participants
Race/Ethnicity, Customized Race · More than one race	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Unknown or not reported	0 Participants n=5 Participants
Region of Enrollment United States	12 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Safety of hCT-MSC Infusion as Measured by Total Number of Infusion Reactions	1 infusion reactions

PRIMARY outcome

Timeframe: 1 year

Population: One participant experienced an infusion reaction.

CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.

Outcome measures

Measure	hCT-MSC Infusion n=1 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Safety of hCT-MSC Infusion as Measured by Severity of Infusion Reactions	1 units on a scale Standard Deviation 0

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Safety of hCT-MSC Infusion as Measured by Total Number of Product-related Infections	0 product-related infections

PRIMARY outcome

Timeframe: 1 year

Population: No participants experienced a product-related infection.

CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 1 year

Population: Three participants were not evaluable due to high levels of pre-existing HLA antibodies and one participant did not have post-infusion data collected.

Outcome measures

Measure	hCT-MSC Infusion n=9 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Safety of hCT-MSC Infusion as Measured by Number of Participants With Evidence of Alloimmunization Via Anti-HLA Antibodies	2 Participants

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Safety of hCT-MSC Infusion as Measured by Total Number of Graft vs. Host Disease (GVHD) Instances	0 instances of GVHD

PRIMARY outcome

Timeframe: 1 year

Population: No participants experienced GVHD.

CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 1 year

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Safety of hCT-MSC Infusion as Measured by Total Number of Unexpected Adverse Events	0 unexpected adverse events

PRIMARY outcome

Timeframe: 1 year

Population: No participants experienced an unexpected adverse event.

CTCAE is used to measure severity: 1= Mild, 2 = Moderate, 3 = Severe, 4 = Life-threatening, 5 = Death.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Baseline, 6 months

6-month T score minus Baseline T score. PDDBI is designed to assess problem behaviors and social, language, and learning or memory skills of children who have been diagnosed with autism spectrum disorder. Raw scores are converted to T scores with a mean of 50 and standard deviation of 10, with a possible range of 10-100. Higher scores represent more severe problem behaviors. Therefore, a negative change in score indicates improvement.

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Change in Pervasive Developmental Disorder Behavior Inventory Autism Composite Score (PDDBI)	-5.00 T score Interval -9.92 to -0.08

PRIMARY outcome

Timeframe: Baseline, 6 months

The change in the average of the Communication and Socialization Standard Subscale Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months. Higher scores indicate greater communication and/or socialization, with a range of 20 to 160 (mean = 100). A positive change in the scores indicates an improvement in communication and/or socialization.

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization and Communication Composite Score	3.83 score on a scale Interval 0.1 to 7.57

PRIMARY outcome

Timeframe: Baseline, 6 months

The number of participants who scored a 1 (very much improved), 2 (much improved), or 3 (minimally improved) on the Clinical Global Impression Scale (CGI-I). The CGI-I score ranges from 1 to 7, where a lower score indicates greater improvement.

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Number of Participants Who Improved (Much Improved or Minimally Improved) on the Clinical Global Impression Scale (CGI)	8 Participants

PRIMARY outcome

Timeframe: Baseline, 6 months

6-month number of words produced minus baseline number of words produced. A positive change indicates improvement.

Outcome measures

Measure	hCT-MSC Infusion n=12 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Change in Communicative Development Inventories (CDI-2)	75.92 words produced Interval 16.1 to 135.73

PRIMARY outcome

Timeframe: Baseline, 6 months

Population: Four participants did not have data available at both time points.

6-month percentage of gaze directed towards social stimulus minus baseline percentage of gaze directed towards social stimulus. A negative change indicates improvement.

Outcome measures

Measure	hCT-MSC Infusion n=8 Participants hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Change in Attention Abilities as Assessed Via Eye-tracking	-0.03 percentage of gaze Interval -0.12 to 0.15

PRIMARY outcome

Timeframe: Baseline, 6 months

Population: Data not collected.

Outcome measures

Outcome data not reported

Adverse Events

hCT-MSC Infusion

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	hCT-MSC Infusion n=12 participants at risk hCT-MSC infusion: This is a single site, phase I, open-label, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in 12 toddlers 18 to 48 months of age with autism spectrum disorder (ASD).
Respiratory, thoracic and mediastinal disorders Upper respiratory infection	66.7% 8/12 • 12 months
Skin and subcutaneous tissue disorders Urticaria	8.3% 1/12 • 12 months
Psychiatric disorders Aggression	8.3% 1/12 • 12 months
Psychiatric disorders Agitation	8.3% 1/12 • 12 months
Immune system disorders Allergic reaction	8.3% 1/12 • 12 months
Gastrointestinal disorders Dental caries	16.7% 2/12 • 12 months
Skin and subcutaneous tissue disorders Eczema	8.3% 1/12 • 12 months
General disorders Fever	25.0% 3/12 • 12 months
General disorders Food Aversions	8.3% 1/12 • 12 months
Gastrointestinal disorders Diarrhea	8.3% 1/12 • 12 months
Psychiatric disorders Hyperactivity	8.3% 1/12 • 12 months
Psychiatric disorders Increased Aggression	8.3% 1/12 • 12 months
Psychiatric disorders Increased Hyperactivity	8.3% 1/12 • 12 months
General disorders Increased Meltdowns	8.3% 1/12 • 12 months
Psychiatric disorders Increased Repetitive Language	8.3% 1/12 • 12 months
Psychiatric disorders Increased Sensory Seeking Behaviors	8.3% 1/12 • 12 months
Infections and infestations Infections and Infestations	8.3% 1/12 • 12 months
Infections and infestations Infections and infestations - Other, specify	8.3% 1/12 • 12 months
General disorders Infusion related reaction	8.3% 1/12 • 12 months
General disorders Meltdowns	8.3% 1/12 • 12 months
General disorders Noncompliance	8.3% 1/12 • 12 months
Ear and labyrinth disorders Otitis media	16.7% 2/12 • 12 months
Psychiatric disorders Repetitive Behaviors	16.7% 2/12 • 12 months
Psychiatric disorders Repetitive Language	8.3% 1/12 • 12 months
Psychiatric disorders Ritualistic Behaviors	16.7% 2/12 • 12 months
Psychiatric disorders Sensory Seeking Behaviors	16.7% 2/12 • 12 months
Skin and subcutaneous tissue disorders Skin infection	8.3% 1/12 • 12 months
Nervous system disorders Sleep Difficulty	16.7% 2/12 • 12 months

Additional Information

Joanne Kurtzberg, MD

Duke University

Phone: 919-668-1119

Email: joanne.kurtzberg@duke.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place