Autism Spectrum DisorderPhase 2MTT-ASDNCT03408886
Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
A Phase 2 study testing Vancomycin, MoviPrep and Full Spectrum Microbiota in people with Autism Spectrum Disorder and Gastrointestinal Disorder, run by Arizona State University at 1 site in United States.
Trial summary
This is a clinical trial of Microbiota Transplant Therapy (MTT) for adults with autism spectrum disorders (ASD) who have gastrointestinal problems. Previous research has shown that individuals with ASD have a low diversity of gut bacteria, and low diversity is generally associated with poor gastrointestinal (GI)… Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Autism Spectrum Disorder, Gastrointestinal Disorder
- What is being tested?
- Vancomycin, MoviPrep and Full Spectrum Microbiota
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Jan 2018to 15 Dec 2024
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adult aged 18-60 years
- Diagnosis of autism per both the Autism Diagnostic Interview - Revised (ADI-R) and the Childhood Autism Rating Scale 2 (CARS-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
- Neurotypical adult observer (such as parent, guardian, or sibling) who observes adult for at least 4 hours/week who can serve as an Evaluator to complete questionnaires on their symptoms with the assistance of the Participant as much as they are able.
- Ability to swallow pills (without chewing)
Who cannot join
- Antibiotics in last 3 months
- 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 14Show fewer
- Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
- Severely underweight/malnourished
- Recent or scheduled surgeries
- Current participation in other clinical trials
- Females who are pregnant or who are sexually active without effective birth control. We will conduct a urine pregnancy test on all female participants as part of the screening and at each clinical visit.
- Allergy or intolerance to vancomycin or MoviPrep
- Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential.
- -
Trial locations and contacts
1 site in 1 country.
About this study
This is a clinical trial of Microbiota Transplant Therapy (MTT) for adults with autism spectrum disorders (ASD) who have gastrointestinal problems.
Previous research has shown that individuals with ASD have a low diversity of gut bacteria, and low diversity is generally associated with poor gastrointestinal (GI) health. We previously found that MTT therapy for children with ASD and GI symptoms was helpful in reducing their GI symptoms, reducing their ASD symptoms, and increasing their diversity of gut bacteria.
This clinical trial will investigate the hypothesis that MTT therapy will be helpful for adults with ASD who have GI symptoms.
Read the detailed description
For adults ages 18-60 years with ASD and gastrointestinal problems, the investigators propose a Phase 2 clinical trial to evaluate the safety and efficacy of MTT. The study will also determine if longer treatment is beneficial, and to conduct a longer observation after treatment stops to determine long-term safety and efficacy. The three parts of this trial are described below.
Part 1: Placebo-Controlled Treatment The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 2-week treatment with oral vancomycin (or placebo), then 1 day of Moviprep to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by oral administration of Full Spectrum Microbiota (FSM) or placebo. An initial high dose of FSM (or placebo) for two days will be followed by a lower maintenance dose of FSM (or placebo) for 8 weeks.
Part 2 Extension and Cross-Over
- For the treatment group from Part 1, there will be an 8-week extension of the maintenance dose, to determine if longer treatment has additional benefits.
- For the placebo group from Part 1, they will receive MoviPrep, an initial high dose of FSM for 2 days, and then a lower dose of FSM for 8 weeks (similar to the treatment group in Part 1, but without the vancomycin). This will help us determine if pre-treatment with vancomycin is needed or not.
Part 3: Follow-up There will be follow-up evaluations at 6, 12, and 18 months after treatment is stopped, to assess long-term efficacy and possible adverse effects.
- Being tested
- Vancomycin, MoviPrep, Full Spectrum Microbiota
- Keywords
- fecal transplant, fecal microbiota transplant, microbiota transplant, vancomycin, moviprep
- Medical subject headings
- Autism Spectrum Disorder, Gastrointestinal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VancomycinDrug
Oral vancomycin is administered to reduce pathogenic bacteria.
MoviPrepDrug
MoviPrep is given at the end of vancomycin therapy to remove the vancomycin and remaining bacteria prior to administering Full-Spectrum Microbiota
Full Spectrum MicrobiotaBiological
Also called fecal microbiota transplant
Gut bacteria from healthy human donors are administered orally in a pill form
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | Group A receives treatment in Part 1 and Part 2 Receives: Drug: Vancomycin; Drug: MoviPrep; Biological: Full Spectrum Microbiota |
| Group B | Other | Group B receives no treatment in Part 1, but does receive treatment in Part 2 Receives: Drug: MoviPrep; Biological: Full Spectrum Microbiota |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks
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: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
Secondary outcomes (3)
Change in Daily Stool Log (DSL) from baseline to 10 weeks
The DSL is a report of the number and type of stools over 14 days. Each stool is rated on a scale of 1-7, where 1=very hard, 4=normal, 7=very soft/liquid. The DSL is scored by the number of days of an abnormal stool (type 1-2 or 6-7) or no stool.
Measured: baseline for 2 weeks; daily for 18 weeks, and 2 weeks at month 10, 16, 22
Change in Social Responsiveness Scale - 2 from baseline to 10 weeks of treatment
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: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; months 10.5, 16.5, 22.5
Change in Aberrant Behavior Checklist from baseline to 10 weeks
a questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors
Measured: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2018
- Study start4 Jan 2018
- Primary completion15 Apr 2022
- Study completion15 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- There are two groups. In Part 1, group A receives treatment and group B receives placebo. In Part 2, group A continues to receive treatment, and group B is switched to treatment.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arizona State University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- James B Adams, PhD, Study director, Arizona State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03408886
- Sponsor's protocol ID
- MTT-Adults-1
- Other IDs
- Wi1XWH-16-1-0492
- Acronym
- MTT-ASD
- Status last verified
- Jan 2023
- First submitted
- 8 Jan 2018
- Last updated
- 9 Jan 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kang DW, Adams JB, Gregory AC, Borody T, Chittick L, Fasano A, Khoruts A, Geis E, Maldonado J, McDonough-Means S, Pollard EL, Roux S, Sadowsky MJ, Lipson KS, Sullivan MB, Caporaso JG, Krajmalnik-Brown R. Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study. Microbiome. 2017 Jan 23;5(1):10. doi: 10.1186/s40168-016-0225-7.
PubMed 28122648
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
- ClinicalTrials.gov ID
- NCT03408886
- Acronym / sponsor ID
- MTT-ASD / MTT-Adults-1
- Keywords
- fecal transplant, fecal microbiota transplant, microbiota transplant, vancomycin, moviprep
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 55 participants (actual)
- Lead sponsor
- Arizona State UniversityUniversity, hospital or other
- Study start
- 4 Jan 2018 (actual)
- Primary completion
- 15 Apr 2022 (actual)
- Study completion
- 15 Dec 2024 (estimated)
- First posted
- 24 Jan 2018 (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.
This is a clinical trial of Microbiota Transplant Therapy (MTT) for adults with autism spectrum disorders (ASD) who have gastrointestinal problems.
Previous research has shown that individuals with ASD have a low diversity of gut bacteria, and low diversity is generally associated with poor gastrointestinal (GI) health. We previously found that MTT therapy for children with ASD and GI symptoms was helpful in reducing their GI symptoms, reducing their ASD symptoms, and increasing their diversity of gut bacteria.
This clinical trial will investigate the hypothesis that MTT therapy will be helpful for adults with ASD who have GI symptoms.
Detailed description
For adults ages 18-60 years with ASD and gastrointestinal problems, the investigators propose a Phase 2 clinical trial to evaluate the safety and efficacy of MTT. The study will also determine if longer treatment is beneficial, and to conduct a longer observation after treatment stops to determine long-term safety and efficacy. The three parts of this trial are described below.
Part 1: Placebo-Controlled Treatment The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 2-week treatment with oral vancomycin (or placebo), then 1 day of Moviprep to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by oral administration of Full Spectrum Microbiota (FSM) or placebo. An initial high dose of FSM (or placebo) for two days will be followed by a lower maintenance dose of FSM (or placebo) for 8 weeks.
Part 2 Extension and Cross-Over
- For the treatment group from Part 1, there will be an 8-week extension of the maintenance dose, to determine if longer treatment has additional benefits.
- For the placebo group from Part 1, they will receive MoviPrep, an initial high dose of FSM for 2 days, and then a lower dose of FSM for 8 weeks (similar to the treatment group in Part 1, but without the vancomycin). This will help us determine if pre-treatment with vancomycin is needed or not.
Part 3: Follow-up There will be follow-up evaluations at 6, 12, and 18 months after treatment is stopped, to assess long-term efficacy and possible adverse effects.
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
- There are two groups. In Part 1, group A receives treatment and group B receives placebo. In Part 2, group A continues to receive treatment, and group B is switched to treatment.
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Drug: Vancomycin, Drug: MoviPrep, Biological: Full Spectrum MicrobiotaGroup A receives treatment in Part 1 and Part 2 |
| Group B | Other | Drug: MoviPrep, Biological: Full Spectrum MicrobiotaGroup B receives no treatment in Part 1, but does receive treatment in Part 2 |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Adult aged 18-60 years
- Diagnosis of autism per both the Autism Diagnostic Interview - Revised (ADI-R) and the Childhood Autism Rating Scale 2 (CARS-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 (14 listed)
- Antibiotics in last 3 months
- 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 |
|---|---|---|
| VancomycinOral vancomycin is administered to reduce pathogenic bacteria. | Drug | Group A |
| MoviPrepMoviPrep is given at the end of vancomycin therapy to remove the vancomycin and remaining bacteria prior to administering Full-Spectrum Microbiota | Drug | Group A, Group B |
| Full Spectrum MicrobiotaAlso: fecal microbiota transplantGut bacteria from healthy human donors are administered orally in a pill form | Biological | Group A, Group B |
Primary outcome
Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks
Time frame: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
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.
3 secondary outcomes
Change in Daily Stool Log (DSL) from baseline to 10 weeks
Time frame: baseline for 2 weeks; daily for 18 weeks, and 2 weeks at month 10, 16, 22
Change in Social Responsiveness Scale - 2 from baseline to 10 weeks of treatment
Time frame: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; months 10.5, 16.5, 22.5
Change in Aberrant Behavior Checklist from baseline to 10 weeks
Time frame: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5
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 9 Jan 2023.
- Background: PubMed 28122648Kang DW, Adams JB, Gregory AC, Borody T, Chittick L, Fasano A, Khoruts A, Geis E, Maldonado J, McDonough-Means S, Pollard EL, Roux S, Sadowsky MJ, Lipson KS, Sullivan MB, Caporaso JG, Krajmalnik-Brown R. Microbiota Transfer Therapy alters gut ecosystem and improves gastrointestinal and autism symptoms: an open-label study. Microbiome. 2017 Jan 23;5(1):10. doi: 10.1186/s40168-016-0225-7.
- link to more information about the research groupLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Autism Spectrum Disorder
RecruitingPhase 1
Cannabidiol (CBD) in Adults With ASD
Autism Spectrum Disorder
RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride for the Treatment of Psychiatric Disorders in Children and Adolescents
Schizophrenia, Autism-Related Irritability
Not yet recruiting
Sensory-integration Therapy Verses Augumented Reality-based Training in ASD
Autism Spectrum Disorder
Recruiting
Cumulative and Booster Effects of Multisession Prefrontal tDCS in ASD Adolescents
Transcranial Direct Current Stimulation, Autistic Spectrum Disorder
Not yet recruiting
"Family Resilience And Mind-body Enhancement" (FRAME): A Randomised Controlled Trial
Autism
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 study director
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, MoviPrep and Full Spectrum Microbiota in people with Autism Spectrum Disorder and Gastrointestinal 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 7 inclusion and 14 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 9 Jan 2023. 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. 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?
Change in Childhood Autism Rating Scale (CARS) from baseline to 10 weeks (time frame: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03408886, which also lists 1 linked publication.
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 9 Jan 2023.