NCT04182633

MTT for Children With ASD Who Have Gastrointestinal Disorders

Microbiota Transfer Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (actual)
• Sponsor: Arizona State University · Academic / Other
• Sex: All
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating children with Autism Spectrum Disorder (ASD) and gastrointestinal problems (primarily constipation and/or diarrhea). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by a bowel cleanse, followed by 12 weeks of Fecal Microbiota (FM).

Detailed description

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.

Conditions

• Autism Spectrum Disorder
• Gastro-Intestinal Disorder
• Constipation
• Diarrhea

Interventions

Type	Name	Description
COMBINATION_PRODUCT	vancomycin, Miralax, intestinal microbiota	vancomycin for 14 days, then Miralax for 1 day, then intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)
COMBINATION_PRODUCT	placebo vancomycin, Miralax, placebo intestinal microbiota	placebo vancomycin for 14 days, then Miralax for 1 day, then placebo intestinal microbiota (high dose for 2 days, then maintenance dose for 12 weeks)

Timeline

• Start date: 2019-11-15
• Primary completion: 2025-02-28
• Completion: 2025-12-30
• First posted: 2019-12-02
• Last updated: 2025-04-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study