MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Completed · Phase 2 · Has a placebo group

Conditions studied: Pitt Hopkins Syndrome

In brief

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).

Key facts

Study ID

NCT04132427

Run by

Arizona State University

People needed

6

Starts

2019-09-30

Expected to finish

2022-04-15

Last updated by the study team

2024-08-26

Who can join

Age: 7 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Arizona State University — Tempe, Arizona, United States

Full record on ClinicalTrials.gov

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