NCT06321796

Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders: MTP-101 P

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Gut-Brain-Axis Therapeutics Inc. · Academic / Other
• Sex: All
• Age: 5 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt-Hopkins Syndrome (PTHS) and gastrointestinal problems (constipation, bloating, abdominal pain). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using magnesium citrate, followed by 4 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 5-17 years old and adults 18-55 years old with Pitt-Hopkins Syndrome (PTHS) and Gastrointestinal (GI) problems who have attempted two standard-of-care (SOC) GI treatments with no alleviation of symptoms. This is a Phase 2 clinical trial that will evaluate the safety, tolerability, and efficacy of a powder version of Microbiota Transfer Therapy (MTT) called MTP-101P. The three parts of this trial are described below. Part 1: Placebo-Controlled Treatment (14 Weeks) The trial will begin with a randomized, double-blind, placebo-controlled trial which will include a 10-day treatment with oral vancomycin (or placebo), then 1 day of magnesium citrate to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by 4 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 vs. Group B: Placebo vancomycin, real magnesium citrate, placebo MTP-101P, real antacid. Part 2: Open-Label Observation and Cross-Over (14 weeks) Group A: Observation over the next 14 weeks (no additional treatment). Group A completes the study at the end of part 2. Group B: They will receive the same treatment that Group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, and an initial high dose of MTP-101P for 4 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 B: There will be a follow-up evaluation 14 weeks post-treatment after the end of Part 2, to assess long-term efficacy and possible adverse effects.

Conditions

• Pitt Hopkins Syndrome

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. Duration: 12.5 Weeks
COMBINATION_PRODUCT	Vancomycin	Oral vancomycin is an antibiotic used for treating C. difficile-associated diarrhea and staphylococcal-induced enterocolitis. Duration: 10 Days
COMBINATION_PRODUCT	Magnesium Citrate	Magnesium citrate is a common over-the-counter laxative and bowel cleanse. Duration: 1 Day
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: 12.5 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: 12.5 weeks
COMBINATION_PRODUCT	Placebo Vancomycin	Same packaging and liquid carrier and flavoring used as for the oral vancomycin, but no vancomycin. Duration: 10 days

Timeline

• Start date: 2024-02-27
• Primary completion: 2025-02-01
• Completion: 2025-05-01
• First posted: 2024-03-20
• Last updated: 2024-03-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study