MedPath

Probiotics and Oxytocin Nasal Spray on Social Behaviors of Autism Spectrum Disorder (ASD) Children

Phase 1
Completed
Conditions
Autism Spectrum Disorder
Interventions
Dietary Supplement: oral probiotics
Dietary Supplement: oral placebo
Registration Number
NCT03337035
Lead Sponsor
Massachusetts General Hospital
Brief Summary

Because oral probiotics reported to potentially induce endogenous Oxytocin, and Oxytocin has been reported to improve social behaviors, the investigators will conduct a pilot trial to compare the effects of probiotics and Oxytocin on social behavioral changes in ASD children. Additionally, the investigators will check oxytocin levels, and perform brain fMRI in some subjects, in order to determine which treatment is more efficient, sustainable, and practical, and whether both treatments in combination are better than either treatment alone. If the trial is conclusive, the investigators will conduct a trial in large scale to understand more the mechanism of ASD behaviors and corresponding effective interventions.

Detailed Description

This study description is in accordance with the Consolidated Standards of Reporting Trials (CONSORT) guidelines that are published for the evaluation of randomized controlled trials. This clinical trial is a randomized, double-blind placebo controlled study. Subjects will be randomized to 2 groups:

Phase 1: a. oral placebo, and b. oral probiotics; Phase 2: a. intranasal Oxytocin(OXT) + oral placebo, and b. intranasal OXT + oral probiotics

The treatment will proceed for a total of 28 weeks. In the first phase (16 weeks), all the patients will be randomly and proportionally divided into two groups: Group A (30 subjects) receives oral probiotics while Group B (30 subjects) receives an oral placebo. In the second phase, subjects in Group A and Group B will continue their respective oral probiotics or placebo administration as in Phase 1. In addition, both groups will be simultaneously administered with intranasal OXT spray.

Testing will be performed 3 times total (before, during, and after treatment). The tests include behavioral surveys, cognitive tests, clinical autonomic tests, and blood tests for oxytocin levels. Investigators plan to select up to 10 subjects from each group to conduct a series of MRI studies at week 0, week 16 and week 28.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
35
Inclusion Criteria
  1. Age between 3-25years old;
  2. Pre-existing diagnosis of autism; subjects may be asked to provide documentation confirming diagnosis by DSM-V-TR criteria, ADOS, ADI-R, or other clinical forms
  3. A care provider who can reliably bring the participant to study visits;
  4. No planned changes in medications or psychosocial interventions during trial (stable medications within the last 2 weeks);
  5. Willingness to provide blood samples
Exclusion Criteria
  1. Pregnant woman (before or during the study).
  2. Comorbidity of other neurological and/or psychiatric disorders such as unstable seizures, schizophrenia, schizoaffective disorder, bipolar disorders or history of substance abuse.
  3. Psychotropic medication use
  4. Subjects with active cardiovascular disease that is not controlled by medication.
  5. Oxytocin, antibiotic, or probiotic use within the last 30 days.
  6. Regular nasal obstruction or nosebleeds
  7. Significant hearing, vision, or motor impairments
  8. Habitual consumption of large volumes of water
  9. Started taking new medications within the last 2 weeks

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
oral probiotics and oxytocin sprayintranasal oxytocinSubjects will receive oral probiotics, 2 pills per day, for 28 weeks. For the last 12 weeks, subjects will also receive intranasal oxytocin spray at the following dose: 4 IU in week 1, 8 IU in week 2, 16 IU in week 3, and 24 IU in weeks 4-12.
oral placebo and oxytocin sprayintranasal oxytocinSubjects will receive oral placebo, 2 pills per day, for 28 weeks. For the last 12 weeks, subjects will also receive intranasal oxytocin spray at the following dose: 4 IU in week 1, 8 IU in week 2, 16 IU in week 3, and 24 IU in weeks 4-12.
oral probiotics and oxytocin sprayoral probioticsSubjects will receive oral probiotics, 2 pills per day, for 28 weeks. For the last 12 weeks, subjects will also receive intranasal oxytocin spray at the following dose: 4 IU in week 1, 8 IU in week 2, 16 IU in week 3, and 24 IU in weeks 4-12.
oral placebo and oxytocin sprayoral placeboSubjects will receive oral placebo, 2 pills per day, for 28 weeks. For the last 12 weeks, subjects will also receive intranasal oxytocin spray at the following dose: 4 IU in week 1, 8 IU in week 2, 16 IU in week 3, and 24 IU in weeks 4-12.
Primary Outcome Measures
NameTimeMethod
Social Responsiveness Scale (SRS) Edition 2change from baseline at 0, 16, and 28 weeks

social communication and behavior - The scoring is based on T-score which is based on the sum of responses as follows (76 to higher - severe, 66 to 75- moderate deficiencies, 60 to 65 - mild deficiencies, 59 and below is not clinically significant for ASD).

Aberrant Behavior Checklist (ABC) Edition 2change from baseline at 0, 16, and 28 weeks

social behavior test - The total score is calculated based on 5 sub-scales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech). There are 58 questions that are scored on a 0-3 scale 0 -"not at all a problem", 1- "the behavior is a problem but slight in degree", 2- "the problem is moderately serious" and 3- "the problem is severe in degree". Based on this sub-scores are calculated and added to get the total score.

Secondary Outcome Measures
NameTimeMethod
Functional MRI (resting state)change from baseline at 0, 16, and 28 weeks

Correlation analysis will be used to calculate the connectivity across different brain regions. T-tests will be used for group comparisons.

Functional MRI (task based)change from baseline at 0, 16, and 28 weeks

General linear modeling will be used to calculate brain responses to the tasks. T-tests will be used for group comparisons.

Autonomic indices 4change from baseline at 0, 16, and 28 weeks

skin electrodermal activity

structural MRIchange from baseline at 0, 16, and 28 weeks

The software Freesurfer will be used to calculate volume of brain regions and diffusion parameters (e.g. fractional anisotropy and mean diffusivity) from structural T1 and diffusion tensor images respectively. T-tests will be applied for group comparisons.

Autonomic indices 1change from baseline at 0, 16, and 28 weeks

Blood volume pulse

Autonomic indices 5change from baseline at 0, 16, and 28 weeks

blood oxygen saturation

Microbiomechange from baseline at 0, 16, and 28 weeks

16s metagenomic sequencing of the microbiome

Neuroinflammation and Oxytocin levelschange from baseline at 0, 16, and 28 weeks

neuroendocrine biomarker measured in blood ( Melatonin, Oxytocin, Tumour Necrosis Factor alpha, testosterone and Interleukin 6)

Autonomic indices 2change from baseline at 0, 16, and 28 weeks

heart rate

Autonomic indices 3change from baseline at 0, 16, and 28 weeks

peripheral skin temperature

Eye tracking and behavioral task (emotion response)change from baseline at 0, 16, and 28 weeks

emotion response - The accuracy and reaction time will be calculated for each time point. Then an ANOVA will be used to compare the results.

Eye tracking and behavioral task (eye behavior)change from baseline at 0, 16, and 28 weeks

eye behavior - Conduct T-tests and calculate P values for total and average fixation values for each area of interest

Eye tracking and Behavioral task (joint attention)change from baseline at 0, 16, and 28 weeks

joint attention - Conduct T-tests and calculate P values for total and average fixation values for each area of interest

Trial Locations

Locations (1)

Massachusetts General Hospital

🇺🇸

Charlestown, Massachusetts, United States

© Copyright 2025. All Rights Reserved by MedPath