Beneficial Bacteria Treatment for Autism

NCT02504554 | Completed Phase 1 Results DMC

• 1 other identifier: STUDY00001053
• Study Type: interventional
• Target: 18
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is an open-label clinical trial to investigate a combination therapy for treating gastrointestinal problems in children with autism spectrum disorders. The combination therapy includes beneficial bacteria.

Conditions

Gastrointestinal Problems; Autism Spectrum Disorders

Keywords

children

Outcome Measures

Primary Outcomes (1)

• Gastrointestinal Symptom Responsiveness Scale (GSRS)

  The GSRS is a measure of gastrointestinal symptoms. It includes 15 questions, rated on a Likert scale of 1-7, from no symptoms to severe symptoms, respectively (i.e., higher is worse). We report an average score for all 15 questions, so the possible range for the average score is also 1-7.

  Baseline and 10 weeks (end of treatment)

Secondary Outcomes (7)

• Parent Global Impressions-Revised (PGI-R)

  Baseline and 10 weeks (end of treatment)

• Blood Safety Markers (Assessment of Blood Chemistry Panel and Complete Blood Count)

  Baseline and 10 weeks (end of treatment)

• Childhood Autism Rating Scale (CARS)

  Baseline and 10 weeks (end of treatment)

• Social Responsiveness Scale (SRS)

  Baseline and 10 weeks (end of treatment)

• Short Sensory Profile

  baseline and 10 weeks

Study Arms (2)

Oral Group

EXPERIMENTAL

This group will receive all treatments orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.

Drug: oral Vancomycin; Drug: MoviPrep; Drug: Prilosec; Biological: human fecal material; processed, frozen administered orally

Rectal Group

EXPERIMENTAL

This group will receive some treatments rectally and some orally. The treatments include a combination of Vancomycin, MoviPrep, Prilosec, and human fecal material; processed, frozen.

Drug: oral Vancomycin; Drug: MoviPrep; Drug: Prilosec; Biological: human fecal material; processed, frozen; administered orally and rectally

Interventions

oral Vancomycin DRUG

an antibiotic

Oral Group; Rectal Group

MoviPrep DRUG

a bowel cleanse

Oral Group; Rectal Group

Prilosec DRUG

a stomach acid suppressan

Oral Group; Rectal Group

human fecal material; processed, frozen administered orally BIOLOGICAL

human fecal material; processed, frozen, administered orally

Oral Group

human fecal material; processed, frozen; administered orally and rectally BIOLOGICAL

human fecal material; processed, frozen; administered orally and rectally

Rectal Group

Eligibility Criteria

• Age: 7 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children ages 7-17 years
• Diagnosis of autism per Autism Diagnostic Interview-Revised (ADI-R)
• Moderate or Severe GI problems ( on the GSRS, a single score of 4 (severe) on any item, or a score of 3 (moderate) on two items, or a score of 2 (mild) or more on any 4 items
• No changes in medications, supplements, diet, therapies, or education in last 3 months, and no intention to change them during clinical trial
• General good physical health aside from gastrointestinal problems
• Cognitive Ability to Provide Informed Assent

You may not qualify if:

• Antibiotics in last 6 months
• Probiotics in last 3 months
• Single-gene disorder (Fragile X, etc.)
• Major brain malformation
• Tube feeding
• Severe gastrointestinal problems that require immediate treatment (life-threatening)
• 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

Sponsors & Collaborators

• Arizona State University: lead
• Northern Arizona University: collaborator
• University of Arizona: collaborator
• University of Minnesota: collaborator

Related Publications (3)

• Kang DW, Adams JB, Vargason T, Santiago M, Hahn J, Krajmalnik-Brown R. Distinct Fecal and Plasma Metabolites in Children with Autism Spectrum Disorders and Their Modulation after Microbiota Transfer Therapy. mSphere. 2020 Oct 21;5(5):e00314-20. doi: 10.1128/mSphere.00314-20.

  PMID: 33087514 RESULT

• Kang DW, Adams JB, Coleman DM, Pollard EL, Maldonado J, McDonough-Means S, Caporaso JG, Krajmalnik-Brown R. Long-term benefit of Microbiota Transfer Therapy on autism symptoms and gut microbiota. Sci Rep. 2019 Apr 9;9(1):5821. doi: 10.1038/s41598-019-42183-0.

  PMID: 30967657 RESULT

• 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.

  PMID: 28122648 RESULT

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Vancomycin; MoviPrep; Omeprazole; Microscopy, Electron, Scanning Transmission; Freezing

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins; 2-Pyridinylmethylsulfinylbenzimidazoles; Sulfoxides; Sulfur Compounds; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Benzimidazoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Microscopy, Electron, Transmission; Microscopy, Electron; Microscopy; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Phase Transition; Physical Phenomena; Cold Temperature; Temperature; Thermodynamics; Chemical Phenomena

Limitations and Caveats

Small study, open-label design, but able to demonstrate safety and possible efficacy. Dosage and duration not optimized, so adjustments in dosage and/or duration may improve results. Therapy involved 4 medications; not clear if all needed.

Results Point of Contact

• Title: James Adams
• Organization: Arizona State University

jim.adams@asu.edu

480 965 3316

Study Officials

• James B Adams, PhD

  Arizona State University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2015
• First Posted: July 22, 2015
• Study Start: July 1, 2014
• Primary Completion: April 1, 2016
• Study Completion: April 1, 2016
• Last Updated: September 12, 2025
• Results First Posted: December 18, 2019

Record last verified: 2025-08