NCT06503978

Microbiota Transplant Therapy for Children With Both Autism Spectrum Disorder and Gastrointestinal Disorders

Dosing Study of Microbiota Transplant Therapy for Children With Autism Spectrum Disorder (ASD) Who Have Gastrointestinal Disorders

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Gut-Brain-Axis Therapeutics Inc. · Academic / Other
• Sex: All
• Age: 4 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Autism-spectrum disorder with Gastrointestinal disorders (constipation, diarrhea, and/or abdominal pain). MTT involves a combination of 14 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using Miralax, followed by 5 days of high dose MTP-101P with an antacid, followed by 12 weeks of a lower maintenance dose of MTP-101P with an antacid.

Detailed description

For children 4 to less than \<18 years old with Autism-spectrum disorder with Gastrointestinal disorders (constipation, diarrhea, and/or abdominal pain). This will be a Phase 2 randomized controlled trial to compare two dosing strategies for microbiota transplant vs. placebo. Participants will be randomly assigned to Group A, B, C or D Part 1: Placebo-Controlled Treatment (15 Weeks) The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 14-day treatment with oral vancomycin (or placebo), then 1 day of Miralax to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by 5 days of initial high dose of MTP-101P taken daily 5 minutes after antacid, and then 12 weeks of a lower maintenance dose of MTP-101P taken daily 5 minutes after an antacid. Group A: Real Treatment. Dose 1 Group B: Real Treatment. Dose 2 Group C: Placebo vancomycin, real Miralax, placebo MTP-101P, real antacid. Group D: Placebo vancomycin, real Miralax, placebo MTP-101P, real antacid. Part 2: Open-Label Observation and Cross-Over (12 weeks) Group A: Observation over the next 12 weeks (no additional treatment). Group B: Observation over the next 12 weeks (no additional treatment). Group A and B completes study at end of part 2. Group C will receive the same dosage group A received in part 1 (Dose 1). This includes 14 days of vancomycin, Miralax, and an initial high dose of MTP-101P for 5 days taken daily 5 minutes after an antacid, and then a lower dose of MTP-101P for 12 weeks taken daily 5 minutes after antacid. Group D will receive the same dosage group B received in part 1 (Dose 2). This includes 14 days of vancomycin, Miralax, and an initial high dose of MTP-101P for 5 days taken daily 5 minutes after an antacid, and then a lower dose of MTP-101P for 12 weeks taken daily 5 minutes after antacid. Part 3: Follow Up Group C and D. There will be a follow-up evaluation 15 weeks post-treatment after the end of Part 2, to assess long-term efficacy and possible adverse effects.

Conditions

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

Interventions

Type	Name	Description
COMBINATION_PRODUCT	MTP-101P	MTP-101P is comprised of standardized dose of total fecal microbiota purified from the stool of healthy donors. Donors are carefully screened via health status questionnaires, physical examinations, reviews of comprehensive medical history, clinical laboratory evaluations, serologic and genomic tests for infectious diseases and metabolic health, and stool-related pathogen testing. The material from the donors is purified to remove the majority of non-bacterial material, washed, lyophilized, and provided to the patient in a sachet contained within a mixing bottle.
COMBINATION_PRODUCT	Vancomycin	Oral vancomycin is an antibiotic used for treating C. difficile-associated diarrhea and staphylococcal-induced enterocolitis. Duration: 14 Days
COMBINATION_PRODUCT	Miralax	Miralax is a common over-the-counter laxative and bowel cleanse
COMBINATION_PRODUCT	Antacid	The antacid will be aluminum/magnesium hydroxide, 1x/day, 5 minutes before the MTP-101P. Each 5 ml consists of Aluminum Hydroxide - 200 mg, Magnesium Hydroxide - 200 mg, and Simethicone - 20 mg. If some participants cannot tolerate the aluminum/magnesium hydroxide, we will allow calcium carbonate alternatives equivalent in acid neutralizing capacity. Duration: 12 weeks
COMBINATION_PRODUCT	Placebo MTP-101P	The same packaging will be used as for MTP-101P, but the sachets will contain a freeze-dried mixture of normal saline containing 10% Trehalose. Duration: 12 weeks
COMBINATION_PRODUCT	Placebo Vancomycin	Same packaging and liquid carrier and flavoring used as for the oral vancomycin, but no vancomycin. Duration: 14 days

Timeline

• Start date: 2024-08-20
• Primary completion: 2025-07-17
• Completion: 2026-08-17
• First posted: 2024-07-16
• Last updated: 2024-09-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study