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Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders (MTT-ASD)

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ClinicalTrials.gov Identifier: NCT03408886
Recruitment Status : Recruiting
First Posted : January 24, 2018
Last Update Posted : August 28, 2019
Sponsor:
Information provided by (Responsible Party):
Arizona State University

Tracking Information
First Submitted Date  ICMJE January 8, 2018
First Posted Date  ICMJE January 24, 2018
Last Update Posted Date August 28, 2019
Actual Study Start Date  ICMJE January 4, 2018
Estimated Primary Completion Date October 4, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 23, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2018)
  • 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 ]
    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.
  • 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 ]
    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.
  • Change in Aberrant Behavior Checklist from baseline to 10 weeks [ Time Frame: baseline; month 2.5, 4.5, 10.5, 16.5, 22.5 ]
    a questionnaire about aberrant behaviors. Scores range from 0 to 174, with higher scores indicating more severe behaviors
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: January 23, 2018)
  • Change in Gastrointestinal Stool and Symptom Questionnaire for Autism, from baseline to 10 weeks [ Time Frame: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5 ]
    a questionnaire about GI-related symptoms.
  • Change in Microbiome composition from baseline to 10 weeks [ Time Frame: baseline; month 1, 2.5, 3.5, 4.5, 10.5, 16.5, 22.5 ]
    analysis of the bacterial composition of stool samples, at the species, genus, and phylum level
  • Change in the Vineland Adaptive Behavior Scale - II, from baseline to 10 weeks [ Time Frame: baseline; month 2.5, 4.5, 22.5 ]
    questionnaire about adaptive behaviors; it yields the developmental age of the participant
  • Change in Ohio State University Clinical Impressions Scale, from baseline 10 weeks [ Time Frame: baseline; month 2.5, 4.5 ]
    assessment of autism symptoms by a clinician. It rates 10 symptoms on a scale of 1-7, with higher scores indicating worse symptoms.
  • Change in Parent Global Impressions, from baseline to 10 weeks [ Time Frame: baseline; weeks 2, 4, 6, 8, 10, 12, 14, 16, 18; month 10.5, 16.5, 22.5 ]
    Assessment of autism symptoms. It rates 20 symptoms on a scale of 1-7, with higher scores indicating worse symptoms
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Official Title  ICMJE Microbiota Transfer Therapy for Adults With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders
Brief 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) 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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 (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
In Part 1, everyone is blinded except for the research pharmacist. In Part 2, the participants and study coordinator are unblinded, but the professional evaluators are blinded.
Primary Purpose: Treatment
Condition  ICMJE
  • Autism Spectrum Disorder
  • Gastrointestinal Disorder
Intervention  ICMJE
  • Drug: Vancomycin
    Oral vancomycin is administered to reduce pathogenic bacteria.
  • Drug: MoviPrep
    MoviPrep is given at the end of vancomycin therapy to remove the vancomycin and remaining bacteria prior to administering Full-Spectrum Microbiota
  • Biological: Full Spectrum Microbiota
    Gut bacteria from healthy human donors are administered orally in a pill form
    Other Name: fecal microbiota transplant
Study Arms  ICMJE
  • Experimental: Group A
    Group A receives treatment in Part 1 and Part 2
    Interventions:
    • Drug: Vancomycin
    • Drug: MoviPrep
    • Biological: Full Spectrum Microbiota
  • Group B
    Group B receives no treatment in Part 1, but does receive treatment in Part 2
    Interventions:
    • Drug: MoviPrep
    • Biological: Full Spectrum Microbiota
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 23, 2018)
84
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 1, 2021
Estimated Primary Completion Date October 4, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Adult aged 18-60 years
  2. Diagnosis of autism per both the Autism Diagnostic Interview - Revised (ADI-R) and the Childhood Autism Rating Scale 2 (CARS-2).
  3. GI disorder as defined below that has lasted for at least 3 years.
  4. No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during the clinical trial.
  5. General good physical health aside from gastrointestinal problems
  6. 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.
  7. Ability to swallow pills (without chewing)

Exclusion Criteria:

  1. Antibiotics in last 3 months
  2. Probiotics in last 2 months, or fecal transplant in last 12 months
  3. Single-gene disorder (Fragile X, etc.)
  4. Major brain malformation
  5. Tube feeding
  6. Severe gastrointestinal problems that require immediate treatment (life-threatening)
  7. Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
  8. Severely underweight/malnourished
  9. Recent or scheduled surgeries
  10. Current participation in other clinical trials
  11. 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.
  12. Allergy or intolerance to vancomycin or MoviPrep
  13. Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel and Complete Blood Count with Differential.

    -

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: James B. Adams, PhD 480 965 3316 jim.adams@asu.edu
Contact: Devon Coleman, BS 480 965 5071 autism@asu.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03408886
Other Study ID Numbers  ICMJE MTT-Adults-1
Wi1XWH-16-1-0492 ( Other Grant/Funding Number: Department of Defense )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Arizona State University
Study Sponsor  ICMJE Arizona State University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: James B Adams, PhD Arizona State University
PRS Account Arizona State University
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP