MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Condition: Pitt Hopkins Syndrome
MeSH terms: Gastrointestinal Diseases; Digestive System Diseases; Hyperventilation; Intellectual Disability; Syndrome; Facies
Study type: Interventional
Phase: Phase 2

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 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.
    

Intervention name(s): vancomycin, magnesium citrate, microbiota; placebo vancomycin, real magnsium citrate, placebo microbiota
Intervention type(s): Combination Product; Combination Product
Intervention description(s): 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; 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
Intervention MeSH terms: Vancomycin; Magnesium citrate; Citric Acid; Sodium Citrate
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Part 1: Randomized, double-blind, placebo-controlled Part 2: Treatment group enters observation, placebo group switched to treatment Part 3: Long-term observation
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: microbiota transfer therapy; fecal transplant; fecal microbiota transplant; intestinal microbiota