Clinical trial NCT04132427: MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Official title: Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pitt Hopkins Syndrome
Keywords: microbiota transfer therapy
fecal transplant
fecal microbiota transplant
intestinal microbiota
Lead sponsor: Arizona State University
Sponsor class: OTHER
Collaborators: name: Pitt Hopkins Research Foundation
First posted: 2019-10-18
Enrollment: 6
Sex: ALL
Minimum age (years): 7
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
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
Primary purpose: TREATMENT

Brief 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 Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT.

Randomized, double-blind, placebo-Controlled Treatment (14 weeks)

The trial will be 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

Eligibility criteria: Inclusion Criteria:

1. Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)
2. GI disorder as defined below that has lasted for at least 2 years.
3. 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.
4. Ability to swallow pills (without chewing)
5. Review of last two years of medical records by the study physician.

Exclusion Criteria:

1. Antibiotics in last 3 months
2. Probiotics in last 2 months, or fecal transplant in last 12 months
3. Tube feeding
4. Severe gastrointestinal problems that require immediate treatment (life-threatening)
5. Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
6. Unstable, poor health (based on study physician's opinion)
7. Recent or scheduled surgeries
8. Current participation in other clinical trials
9. Females who are pregnant or who are at risk of pregnancy and sexually active without effective birth control.
10. Allergy or intolerance to vancomycin or magnesium citrate
11. Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel, and Complete Blood Count with Differential.
12. 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.

Primary outcomes:
measure: Daily Stool Record (DSR(
timeFrame: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)
measure: Safety Measures
timeFrame: weeks 0-14
Secondary outcomes:
measure: CGI for GI Disorders
timeFrame: change in score between baseline and week 14
measure: CGI for PTHS Symptoms
timeFrame: change in score between baseline and week 14
measure: PGI-PTHS
timeFrame: change in score between baseline and week 14
measure: GSRS
timeFrame: change in score between baseline and week 14
measure: FLACC
timeFrame: change in score between baseline and week 14
Other outcomes:
Locations:
city: Tempe
country: United States
facility: Arizona State University
state: Arizona
Study officials:
References: