Autism Spectrum DisorderPhase 2NCT04182633
MTT for Children With ASD Who Have Gastrointestinal Disorders
A Phase 2 study testing vancomycin, Miralax, intestinal microbiota in people with Autism Spectrum Disorder and Gastro-Intestinal Disorder, run by Arizona State University at 1 site in United States.
Trial summary
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating children with Autism Spectrum Disorder (ASD) and gastrointestinal problems (primarily constipation and/or diarrhea). Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Autism Spectrum Disorder, Gastro-Intestinal Disorder, Constipation
- What is being tested?
- vancomycin, Miralax, intestinal microbiota
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Nov 2019to 30 Dec 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Child aged 5-17 years
- Diagnosis of autism per the Childhood Autism Rating Scale 2 (CARS-2) and either the Autism Diagnostic Interview-Revised (ADI-R) or the Autism Diagnostic Observation Schedule 2 (ADOS 2).
- GI disorder as defined below that has lasted for at least 3 years.
- No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during the clinical trial.
- General good physical health aside from gastrointestinal problems
- Ability to swallow pills (without chewing)
Who cannot join
- Antibiotics in last 3 months (does not apply to topical antibiotics)
- Probiotics in last 2 months, or fecal transplant in last 12 months
- Single-gene disorder (Fragile X, etc.)
- Major brain malformation
- Tube feeding
- Severe gastrointestinal problems that require immediate treatment (life-threatening)
Show all 16Show fewer
- Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
- Severely underweight/malnourished (per physician clinical judgement)
- Unstable, poor health; seizure disorder that is not responsive to treatment or not on stable management or complex type; or other health conditions that would significantly increase their risk of adverse effects (per physician clinical judgement)
- Recent or scheduled surgeries
- Current participation in other clinical trials
- Females who are pregnant or who are sexually active without effective birth control.
- Allergy or intolerance to vancomycin or Miralax
- Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential.
- Psychotropic medication daily use - Current or within past 2 months - which are known to interfere in gastrointestinal function
- Evidence of significant impairment of immune system, or taking medications that can compromise the immune system, and thus increase risk if exposed to multiple-drug resistant bacteria.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating children with Autism Spectrum Disorder (ASD) and gastrointestinal problems (primarily constipation and/or diarrhea). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by a bowel cleanse, followed by 12 weeks of Fecal Microbiota (FM).
Read the detailed description
For children ages 5-17 years with Autism Spectrum Disorder and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT. The three parts of this trial are described below.
Part 1: Randomized Trial The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 2-week treatment with oral vancomycin (Group A) or placebo (Group B), then 1 day of Miralax to cleanse the bowel of vancomycin and bacteria/feces (both groups), followed by oral administration of FM (Group A) or placebo (Group B). An initial high dose of FM (or placebo) for two days will be followed by a lower maintenance dose of FM or placebo for 12 weeks.
Part 2: Extension (Group A) and Treatment (Group B)
- For Group A there will be 12-week observation period
- For Group B, there will be treatment similar to that received by the treatment group in Part 1 (partial cross-over, open-label), except that they will not receive vancomycin. So, they will be treated with Miralax followed by an initial high dose of FM for 2 days, and then a maintenance dose of FM for 12 weeks. The purpose of this arm is to determine if vancomycin is necessary or not.
Part 3: Follow-up There will be a follow-up evaluation at 6, 12, and 18 months after treatment ends, to determine long-term safety and efficacy.
Participant duration will be approximately 6.5 months for treatment, and follow-up at 6, 12 and 18 months after treatment ends.
Study duration will be approximately 4 years, including IRB approvals, training staff, recruiting, clinical trial, data analysis, and reporting.
- Being tested
- vancomycin, Miralax, intestinal microbiota, placebo vancomycin, Miralax, placebo intestinal microbiota
- Keywords
- fecal transplant, fecal microbiota transplant, intestinal microbiota transplant, microbiota transplant, autism, autism spectrum disorder
- Medical subject headings
- Autism Spectrum Disorder, Digestive System Diseases, Constipation, Diarrhea, Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vancomycin, Miralax, intestinal microbiotaCombination product
vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)
placebo vancomycin, Miralax, placebo intestinal microbiotaCombination product
placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)
| Study group | Role | What they receive |
|---|---|---|
| Group A - Treatment Group | Experimental | This group will receive vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota for 2 days at high dose, then intestinal microbiota for 12 weeks at a maintenance dose Receives: Combination Product: vancomycin, Miralax, intestinal microbiota |
| Group B - Control Group (Miralax only for 1 day) | Placebo comparator | This group will receive placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota for 2 days at high dose, then placebo intestinal microbiota for 12 weeks at a maintenance dose Receives: Combination Product: placebo vancomycin, Miralax, placebo intestinal microbiota |
What is being measured
The primary outcome is the main question the study is designed to answer.
Childhood Autism Rating Scale (CARS)
An evaluation by a trained evaluator of autism symptoms. The range is 15-60, and scores in the range of 27-30 or higher are indicative of autism.
Measured: change in score from baseline to 14 weeks (end of treatment)
Gastrointestinal Symptom Severity Scale (GSRS)
A 15-item questionnaire for evaluation of GI symptoms, with each item rated on a scale of 1 (no symptoms) to 7 (severe symptoms), with an average score of 1-7.
Measured: change in score from baseline to 14 weeks (end of treatment)
Secondary outcomes (3)
Daily Stool Record
The DSR is a daily record of bowel movements including Bristol Stool Form scale. It is rated as the % of days with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.
Measured: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)]
Social Responsiveness Scale 2
A questionnaire about social skills, where higher scores suggest more severe autism. Scores range from 0 to 195, with higher scores indicating more severe problems.
Measured: change in score from baseline to 14 weeks (end of treatment)
Aberrant Behavior Checklist
A questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors
Measured: change in score from baseline to 14 weeks (end of treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2019
- Study start15 Nov 2019
- Primary completion28 Feb 2025
- Study completion30 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Arizona State UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a randomized, double-blind, placebo-controlled study.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arizona State University
- Sponsor type
- University, hospital or other
- Collaborators
- University of Minnesota
- Responsible party
- Sponsor
- Investigators
- James B Adams, PhD, Principal investigator, Arizona State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04182633
- Sponsor's protocol ID
- NEIRB#: 120190306
- Status last verified
- Apr 2025
- First submitted
- 27 Nov 2019
- Last updated
- 25 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
- ClinicalTrials.gov ID
- NCT04182633
- Acronym / sponsor ID
- NEIRB#: 120190306
- Keywords
- fecal transplant, fecal microbiota transplant, intestinal microbiota transplant, microbiota transplant, autism, autism spectrum disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Arizona State UniversityUniversity, hospital or other
- Collaborators
- University of Minnesota
- Study start
- 15 Nov 2019 (actual)
- Primary completion
- 28 Feb 2025 (actual)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 2 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating children with Autism Spectrum Disorder (ASD) and gastrointestinal problems (primarily constipation and/or diarrhea). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by a bowel cleanse, followed by 12 weeks of Fecal Microbiota (FM).
Detailed description
For children ages 5-17 years with Autism Spectrum Disorder and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT. The three parts of this trial are described below.
Part 1: Randomized Trial The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 2-week treatment with oral vancomycin (Group A) or placebo (Group B), then 1 day of Miralax to cleanse the bowel of vancomycin and bacteria/feces (both groups), followed by oral administration of FM (Group A) or placebo (Group B). An initial high dose of FM (or placebo) for two days will be followed by a lower maintenance dose of FM or placebo for 12 weeks.
Part 2: Extension (Group A) and Treatment (Group B)
- For Group A there will be 12-week observation period
- For Group B, there will be treatment similar to that received by the treatment group in Part 1 (partial cross-over, open-label), except that they will not receive vancomycin. So, they will be treated with Miralax followed by an initial high dose of FM for 2 days, and then a maintenance dose of FM for 12 weeks. The purpose of this arm is to determine if vancomycin is necessary or not.
Part 3: Follow-up There will be a follow-up evaluation at 6, 12, and 18 months after treatment ends, to determine long-term safety and efficacy.
Participant duration will be approximately 6.5 months for treatment, and follow-up at 6, 12 and 18 months after treatment ends.
Study duration will be approximately 4 years, including IRB approvals, training staff, recruiting, clinical trial, data analysis, and reporting.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This is a randomized, double-blind, placebo-controlled study.
| Arm | Type | Receives |
|---|---|---|
| Group A - Treatment Group | Experimental | Combination Product: vancomycin, Miralax, intestinal microbiotaThis group will receive vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota for 2 days at high dose, then intestinal microbiota for 12 weeks at a maintenance dose |
| Group B - Control Group (Miralax only for 1 day) | Placebo comparator | Combination Product: placebo vancomycin, Miralax, placebo intestinal microbiotaThis group will receive placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota for 2 days at high dose, then placebo intestinal microbiota for 12 weeks at a maintenance dose |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Child aged 5-17 years
- Diagnosis of autism per the Childhood Autism Rating Scale 2 (CARS-2) and either the Autism Diagnostic Interview-Revised (ADI-R) or the Autism Diagnostic Observation Schedule 2 (ADOS 2).
- GI disorder as defined below that has lasted for at least 3 years.
- No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during the clinical trial.
- General good physical health aside from gastrointestinal problems
Key exclusion criteria (16 listed)
- Antibiotics in last 3 months (does not apply to topical antibiotics)
- Probiotics in last 2 months, or fecal transplant in last 12 months
- Single-gene disorder (Fragile X, etc.)
- Major brain malformation
- Tube feeding
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vancomycin, Miralax, intestinal microbiotavancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks) | Combination product | Group A - Treatment Group |
| placebo vancomycin, Miralax, placebo intestinal microbiotaplacebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks) | Combination product | Group B - Control Group (Miralax only for 1 day) |
Primary outcome
Childhood Autism Rating Scale (CARS)
Time frame: change in score from baseline to 14 weeks (end of treatment)
An evaluation by a trained evaluator of autism symptoms. The range is 15-60, and scores in the range of 27-30 or higher are indicative of autism.
Gastrointestinal Symptom Severity Scale (GSRS)
Time frame: change in score from baseline to 14 weeks (end of treatment)
A 15-item questionnaire for evaluation of GI symptoms, with each item rated on a scale of 1 (no symptoms) to 7 (severe symptoms), with an average score of 1-7.
3 secondary outcomes
Daily Stool Record
Time frame: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)]
Social Responsiveness Scale 2
Time frame: change in score from baseline to 14 weeks (end of treatment)
Aberrant Behavior Checklist
Time frame: change in score from baseline to 14 weeks (end of treatment)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Apr 2025.
- The web page for the research group leading this studyLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James B Adams, PhD principal investigator
Arizona State University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing vancomycin, Miralax, intestinal microbiota in people with Autism Spectrum Disorder and Gastro-Intestinal Disorder, run by Arizona State University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 25 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Childhood Autism Rating Scale (CARS) (time frame: change in score from baseline to 14 weeks (end of treatment)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Apr 2025.