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Clinical Trial 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).


Clinical Trial Description

For children ages 5-17 years with PTHS and gastrointestinal problems, a 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: 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 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 Part 2 Open-Label Observation and Cross-Over (14 weeks) Group 1: Observation over the next 14 weeks (no additional treatment) Group 2: They will receive the same treatment that group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, an initial high dose of FM for 4 days, and then a lower dose of FM for 12 weeks. Part 3: Follow-up There will be a follow-up evaluation at 14 weeks after the end of part 2, to assess long-term efficacy and possible adverse effects. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04132427
Study type Interventional
Source Arizona State University
Contact
Status Completed
Phase Phase 2
Start date September 30, 2019
Completion date April 15, 2022

See also
  Status Clinical Trial Phase
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Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting NCT06321796 - Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders Phase 2
Not yet recruiting NCT05165017 - Safety & Efficacy of AlloRx SC® in PTHS Patients Phase 1/Phase 2