NCT04132427

MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Arizona State University · Academic / Other
• Sex: All
• Age: 7 Years – 17 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 similar to Irritable Bowel Syndrome (IBS). 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 PTHS and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT. Randomized, double-blind, placebo-Controlled Treatment (14 weeks) The trial will be 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 oral administration of FM (or placebo). 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. Group A: real treatment Group B: placebo vancomycin, real magnesium citrate, placebo FM

Conditions

• Pitt Hopkins Syndrome

Interventions

Type	Name	Description
COMBINATION_PRODUCT	vancomycin, magnesium citrate, microbiota	10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota
COMBINATION_PRODUCT	placebo vancomycin, real magnesium citrate, placebo microbiota	10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota

Timeline

• Start date: 2019-09-30
• Primary completion: 2022-03-17
• Completion: 2022-04-15
• First posted: 2019-10-18
• Last updated: 2024-08-26
• Results posted: 2024-08-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study