MTT for Children With ASD Who Have Gastrointestinal Disorders

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.

Inclusion criteria

for Children with ASD

• 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)

Exclusion criteria

for Children with ASD

• 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)
• 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.