Phase 2
N=6
MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Pitt Hopkins Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04132427 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Aug 2024
Primary outcome: Primary: Daily Stool Record (DSR( — -2.3; 0 days
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- vancomycin, magnesium citrate, microbiota (Combination_product); placebo vancomycin, real magnesium citrate, placebo microbiota (Combination_product)
- Age
- Pediatric · 7+ yrs
- Sex
- All
- Sponsor
- Arizona State University
- Primary completion
- Mar 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Daily Stool Record (DSR( |
-2.3; 0 | — |
| PRIMARY Safety Measures |
8; 10 | — |
| SECONDARY CGI for GI Disorders |
-1; -.67 | — |
| SECONDARY CGI for PTHS Symptoms |
0; -.33 | — |
| SECONDARY PGI-PTHS |
.68; .29 | — |
| SECONDARY GSRS |
-2.3; -1.07 | — |
| SECONDARY FLACC |
-4.0; -2.67 | — |
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).
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT04132427). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.