Trial | NCT04132427  Report issue

Title

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

  • Phase

    Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Study Participants

    7
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).
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 Started
Sep 30
2019
Primary Completion
Mar 17
2022
Study Completion
Apr 15
2022
Last Update
Jan 09
2023

Combination Product vancomycin, magnesium citrate, microbiota [vancomycin (Vancocin), human fecal microbiota (Reybota), magnesium citrate]

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 magnsium 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

Group A: Treatment Experimental

Vancomycin, magnesium citrate, microbiota

Group B: Placebo Placebo Comparator

placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota

Criteria 

Inclusion Criteria:

Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)
GI disorder as defined below that has lasted for at least 2 years.
No changes in medications, supplements, diet, or therapies in last 2 months, and no intention to change them during the Parts 1 and 2 of the clinical trial.
Ability to swallow pills (without chewing)
Review of last two years of medical records by the study physician.

Exclusion Criteria:

Antibiotics in last 3 months
Probiotics in last 2 months, or fecal transplant in last 12 months
Tube feeding
Severe gastrointestinal problems that require immediate treatment (life-threatening)
Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
Unstable, poor health (based on study physician's opinion)
Recent or scheduled surgeries
Current participation in other clinical trials
Females who are pregnant or who are at risk of pregnancy and sexually active without effective birth control.
Allergy or intolerance to vancomycin or magnesium citrate
Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel, and Complete Blood Count with Differential.
Evidence of significant impairment of immune system, or taking medications that can compromise the immune system, and thus increase risk if exposed to multiple-drug resistant bacteria.
No Results Posted