Autism Spectrum DisordersEarly Phase 1NCT02903030
Probiotics for Quality of Life in Autism Spectrum Disorders
A Early Phase 1 study testing Visbiome Extra Strength in people with Autism Spectrum Disorders and Anxiety, run by Ohio State University at 1 site in United States.
Trial summary
A randomized pilot trial of a probiotic for quality of life in autism spectrum disorder (ASD), targeting gastrointestinal (GI) symptoms. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Autism Spectrum Disorders, Anxiety
- What is being tested?
- Visbiome Extra Strength
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2016to 1 Dec 2017
- Phase
- Early Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- have DSM-5 ASD on clinical evaluation by a doctoral-level diagnostician, confirmed by Autism Diagnostic Interview-Revised or Autism Diagnostic Observation Schedule;
- be between 3 and 12 years old;
- have >2 mo. abdominal pain, constipation, diarrhea, and/or vomiting, with an item-mean score >2 on at least one scale of the GI module of the PedsQL scale;
- have clinical anxiety symptoms with an item mean of >1.0 (0-3 scale) on the new Autism Anxiety Scale.
- Participants will be recruited from minority, poor, inner city, or rural populations.
Who cannot join
- Antibiotics in 2 months prior to enrolling;
- Prior bowel surgery;
- Chronic serious medical condition (e.g., diabetes);
- Weight or height < 3rd %ile for age;
- Chronic anti-inflammatory use within 2 months prior to enrolling;
- History of inflammatory bowel disease, Celiac disease, or eosinophilic disorders (e.g., eosinophilic esophagitis);
- Already taking probiotics within the previous 6 months.
Trial locations and contacts
1 site in 1 country.
United States1 site
Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
About this study
A randomized pilot trial of a probiotic for quality of life in autism spectrum disorder (ASD), targeting gastrointestinal (GI) symptoms.
Read the detailed description
The physical and mental/emotional health of people with autism spectrum disorder (ASD) are closely connected. The emerging data on immune abnormalities and gut microbiome differences, and interactions of the genome with these suggest a possible etiological link between physical and mental dysfunction, especially the gastrointestinal (GI) dysfunction and severe anxiety that many individuals with ASD manifest. The investigators have preliminary clinical evidence that children with ASD \& GI symptoms differ in microbiome composition and function from neurotypical children with GI symptoms. The investigators hypothesize that altered host-microbial signals, which include altered fecal neurotransmitter gamma-aminobutyric acid (GABA) levels contribute towards anxiety and sensory over-responsivity in ASD. Our preliminary findings also show that probiotic Visbiome Extra Strength, improves GI and pain symptoms, correlating with altered gut microbiome composition and related metabolites (the macrobiome). The proposed crossover trial will explore the possibilities of this new appreciation of the microbiome-mental/physical function connection for ASD, GI dysfunction, and anxiety. If altering the gut microbiome results in better GI and emotional function, it could improve the quality of life for children with ASD and their parents. A pilot trial with 12 children with ASD will test feasibility for a proposed three-site crossover randomized clinical trial (RCT) of probiotics (beneficial bacteria including Lactobacilli \& Bifidobacteria) in 60 children 3-12 years old with ASD, GI dysfunction, \& anxiety. In a balanced crossover children will be randomized 1;1 to Visbiome or placebo first, 8 weeks per condition with 3 weeks washout between. The investigators have access to significant fecal microbiome and metabolome data from NIH-funded Human Microbiome Projects (HMP) on similar-age healthy and irritable-bowel children, with and without ASD. These will help leverage our understanding of macrobiome changes that correlate with functional improvement of GI and abdominal pain symptoms. Pilot study efficiency will also benefit from those HMPs having already collected and analyzed baseline stools for some children with ASD, thus saving significant costs for baseline stool analyses for the pilot.
- Conditions
- Autism Spectrum Disorders, Anxiety
- Being tested
- Maltose (placebo), Visbiome Extra Strength
- Keywords
- Gastrointestinal Microbiome
- Medical subject headings
- Autism Spectrum Disorder, Anxiety Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Maltose (placebo)Drug
Also called Maltose with silicon dioxide
Maltose with a trace amount of silicon dioxide
Visbiome Extra StrengthDrug
It is a mix of 8 strains of beneficial bacteria that should improve gut flora, improving GI function and hopefully anxiety
| Study group | Role | What they receive |
|---|---|---|
| Visbiome, Then Placebo | Experimental | The probiotic mix (VISBIOME) will be mainly Bifidobacteria and Lactobacilli, in view of the previously reported encouraging clinical studies and safety data. Receives: Drug: Visbiome Extra Strength |
| Placebo, Then Visbiome | Placebo comparator | Placebo matched to probiotic. Receives: Drug: Maltose (placebo) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Gastrointestinal (GI) Module of the Pediatric Quality of Life Inventory (PedsQL) at From Baseline at Week 8
A 74-item survey with 14 scales. Report forms for specific age ranges assess the parent's perception of the child's GI function and/or symptoms during the last month on a 5-point scale from 0 (never a problem) to 4 (almost always a problem). Items are reverse-scored and transformed to a 0-100 scale so lower scores reflect worse GI dysfunction. Response choices are in Likert-scale format ranging from 0 to 4 (0=Never, 1=Almost Never, 2=Sometimes, 3=Often, 4=Almost Always).
Measured: Baseline and Week 8 of Both the First and Second Intervention
Secondary outcomes (6)
Change in Target Symptom Rating From Baseline at Week 8
Parents are asked to name the 2 problems of most concern to them at baseline; a clinician helps the parent quantify and describe the problem (frequency, duration, severity, interference with daily life) at baseline. At subsequent visits the clinician reminds the parent of the previous description and helps them again quantify/describe the current state. A panel of blind clinicians reviews the descriptions and rates each on a 9-point scale relative to baseline, from remission (0) to disastrously worse (9), with 5=no change. These ratings are averaged, capturing the issues of most concern to parents across families. For purposes of this study, one of the 2 problems will be required to pertain to GI function, and will be analyzed separately as well as being averaged into the overall symptom rating.
Measured: Baseline and Week 8 of Both the First and Second Intervention
Change in Parent Anxiety Checklist--ASD From Baseline at Week 8
A 25-item single-factor scale measure of emotional stability/anxiety. Higher scores are worse for PRAS-ASD subscale, hence a negative estimate means more improvement with the probiotics compared to control. Scores range from 0-75.
Measured: Baseline and Week 8 of Both the First and Second Intervention
Change in The Aberrant Behavior Checklist (ABC) From Baseline at Week 8
The ABC is a 58-item parent rating on a 0-3 scale with five subscales:
- Irritability (includes agitation, aggression, and self-injury, 15 items) with range of scores from 0-45
- Social Withdrawal (16 items) with range of scores from 0-48
- Stereotypies (7 items) with range of scores from 0-21
- Hyperactivity (16 items) with range of scores from 0-48
- Inappropriate Speech (4 items) with range of scores from 0-12.
Higher scores are worse for the ABC subscale, hence a negative estimate means more improvement with the probiotics compared to control.
Measured: Baseline and Week 8 of Both the First and Second Intervention
Change in Social Responsiveness Scale (SRS) From Baseline at Week 8
This 65-item rating scale measures the severity of autism spectrum symptoms as they occur in natural social settings. The SRS provides a clear picture of a child's social impairments, assessing social awareness, social information processing, capacity for reciprocal social communication, social anxiety/avoidance, and autistic preoccupations and traits. It is appropriate for use with children from 4 to 18 years of age and will detect changes in core ASD symptoms. A higher score in this scale represents worse symptoms with raw scores summed and generated t-scores ranging from 0-110.
Measured: Baseline and Week 8 of Both the First and Second Intervention
Children's Sleep Habits Questionnaire (CSHQ) at Week 8
It includes 33 items rated retrospectively over the previous week by parents yielding a total score and eight subscales. Eight subscales include: (1) bedtime resistance (2) sleep onset latency, (3) sleep duration, (4) anxiety around sleep, (5) night awakenings, (6) sleep disordered breathing, (7) parasomnias and (8) morning waking/daytime sleepiness.Total score (summed) range of 0-99. A higher score is a better outcome for this measure.
Measured: Week 8 of Both the First and Second Intervention
Change in The Parenting Stress Index Short Form (PSI)
The PSI is used to evaluate the degree of stress in the parent-child relationship. The Short Form has 36 items from the full length PSI, rated on a 5-point scale from 1 = strongly disagree, to 5 = strongly agree. It is completed in 10-15 minutes. The PSI may be used for parents of children up to 12 years. It yields a Total Score and three domain scores. This will detect effect on parental stress and QOL. Higher scores (ranging from 0-180) are worse for PSI, hence a negative estimate means more improvement with the probiotics compared to control.
Measured: Baseline and Week 8 of Both the First and Second Intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2016 (estimated)
- Study startAug 2016
- Primary completion1 Dec 2017
- Study completion1 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Autism Spectrum DisordersStudies of the same condition, open and under way.
- More trials run by Ohio 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ohio State University
- Sponsor type
- University, hospital or other
- Collaborators
- Autism Treatment Network, Autism Speaks
- Responsible party
- Principal investigator, L. Eugene Arnold, Ohio State University
- Investigators
- Eugene Arnold, MD, Principal investigator, Ohio State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02903030
- Sponsor's protocol ID
- 2016H0174
- Status last verified
- Jan 2020
- First submitted
- 14 Jun 2016
- Last updated
- 5 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Probiotics for Quality of Life in Autism Spectrum Disorders
- ClinicalTrials.gov ID
- NCT02903030
- Acronym / sponsor ID
- 2016H0174
- Conditions
- Autism Spectrum Disorders, Anxiety
- Keywords
- Gastrointestinal Microbiome
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Ohio State UniversityUniversity, hospital or other
- Collaborators
- Autism Treatment Network, Autism Speaks
- Study start
- Aug 2016
- Primary completion
- 1 Dec 2017 (actual)
- Study completion
- 1 Dec 2017 (actual)
- First posted
- 16 Sep 2016 (estimated)
- Results
- Posted 5 Feb 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A randomized pilot trial of a probiotic for quality of life in autism spectrum disorder (ASD), targeting gastrointestinal (GI) symptoms.
Detailed description
The physical and mental/emotional health of people with autism spectrum disorder (ASD) are closely connected. The emerging data on immune abnormalities and gut microbiome differences, and interactions of the genome with these suggest a possible etiological link between physical and mental dysfunction, especially the gastrointestinal (GI) dysfunction and severe anxiety that many individuals with ASD manifest. The investigators have preliminary clinical evidence that children with ASD \& GI symptoms differ in microbiome composition and function from neurotypical children with GI symptoms. The investigators hypothesize that altered host-microbial signals, which include altered fecal neurotransmitter gamma-aminobutyric acid (GABA) levels contribute towards anxiety and sensory over-responsivity in ASD. Our preliminary findings also show that probiotic Visbiome Extra Strength, improves GI and pain symptoms, correlating with altered gut microbiome composition and related metabolites (the macrobiome). The proposed crossover trial will explore the possibilities of this new appreciation of the microbiome-mental/physical function connection for ASD, GI dysfunction, and anxiety. If altering the gut microbiome results in better GI and emotional function, it could improve the quality of life for children with ASD and their parents. A pilot trial with 12 children with ASD will test feasibility for a proposed three-site crossover randomized clinical trial (RCT) of probiotics (beneficial bacteria including Lactobacilli \& Bifidobacteria) in 60 children 3-12 years old with ASD, GI dysfunction, \& anxiety. In a balanced crossover children will be randomized 1;1 to Visbiome or placebo first, 8 weeks per condition with 3 weeks washout between. The investigators have access to significant fecal microbiome and metabolome data from NIH-funded Human Microbiome Projects (HMP) on similar-age healthy and irritable-bowel children, with and without ASD. These will help leverage our understanding of macrobiome changes that correlate with functional improvement of GI and abdominal pain symptoms. Pilot study efficiency will also benefit from those HMPs having already collected and analyzed baseline stools for some children with ASD, thus saving significant costs for baseline stool analyses for the pilot.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Visbiome, Then Placebo | Experimental | Drug: Visbiome Extra StrengthThe probiotic mix (VISBIOME) will be mainly Bifidobacteria and Lactobacilli, in view of the previously reported encouraging clinical studies and safety data. |
| Placebo, Then Visbiome | Placebo comparator | Drug: Maltose (placebo)Placebo matched to probiotic. |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- have DSM-5 ASD on clinical evaluation by a doctoral-level diagnostician, confirmed by Autism Diagnostic Interview-Revised or Autism Diagnostic Observation Schedule;
- be between 3 and 12 years old;
- have >2 mo. abdominal pain, constipation, diarrhea, and/or vomiting, with an item-mean score >2 on at least one scale of the GI module of the PedsQL scale;
- have clinical anxiety symptoms with an item mean of >1.0 (0-3 scale) on the new Autism Anxiety Scale.
- Participants will be recruited from minority, poor, inner city, or rural populations.
Key exclusion criteria (7 listed)
- Antibiotics in 2 months prior to enrolling;
- Prior bowel surgery;
- Chronic serious medical condition (e.g., diabetes);
- Weight or height < 3rd %ile for age;
- Chronic anti-inflammatory use within 2 months prior to enrolling;
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 |
|---|---|---|
| Maltose (placebo)Also: Maltose with silicon dioxideMaltose with a trace amount of silicon dioxide | Drug | Placebo, Then Visbiome |
| Visbiome Extra StrengthIt is a mix of 8 strains of beneficial bacteria that should improve gut flora, improving GI function and hopefully anxiety | Drug | Visbiome, Then Placebo |
Primary outcome
Change in Gastrointestinal (GI) Module of the Pediatric Quality of Life Inventory (PedsQL) at From Baseline at Week 8
Time frame: Baseline and Week 8 of Both the First and Second Intervention
A 74-item survey with 14 scales. Report forms for specific age ranges assess the parent's perception of the child's GI function and/or symptoms during the last month on a 5-point scale from 0 (never a problem) to 4 (almost always a problem). Items are reverse-scored and transformed to a 0-100 scale so lower scores reflect worse GI dysfunction. Response choices are in Likert-scale format ranging from 0 to 4 (0=Never, 1=Almost Never, 2=Sometimes, 3=Often, 4=Almost Always).
6 secondary outcomes
Change in Target Symptom Rating From Baseline at Week 8
Time frame: Baseline and Week 8 of Both the First and Second Intervention
Change in Parent Anxiety Checklist--ASD From Baseline at Week 8
Time frame: Baseline and Week 8 of Both the First and Second Intervention
Change in The Aberrant Behavior Checklist (ABC) From Baseline at Week 8
Time frame: Baseline and Week 8 of Both the First and Second Intervention
Change in Social Responsiveness Scale (SRS) From Baseline at Week 8
Time frame: Baseline and Week 8 of Both the First and Second Intervention
Children's Sleep Habits Questionnaire (CSHQ) at Week 8
Time frame: Week 8 of Both the First and Second Intervention
Change in The Parenting Stress Index Short Form (PSI)
Time frame: Baseline and Week 8 of Both the First and Second Intervention
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Eugene Arnold, MD principal investigator
Ohio State University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Visbiome Extra Strength in people with Autism Spectrum Disorders and Anxiety, run by Ohio 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 5 inclusion and 7 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: finished, as of the sponsor's last update on 5 Feb 2020. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Feb 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Gastrointestinal (GI) Module of the Pediatric Quality of Life Inventory (PedsQL) at From Baseline at Week 8 (time frame: Baseline and Week 8 of Both the First and Second Intervention). 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 5 Feb 2020.