MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Arizona State University (ASU)

Status and phase: Completed; Phase 2

Conditions: Pitt Hopkins Syndrome

Treatments: Combination Product: placebo vancomycin, real magnesium citrate, placebo microbiota; Combination Product: vancomycin, magnesium citrate, microbiota

Study type: Interventional

Funder types: Other

Identifiers

NCT04132427

IRB#: 120190263

Details and patient eligibility

About

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).

Full 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

Enrollment: 6 patients

Sex: All

Ages: 7 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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6 participants in 2 patient groups, including a placebo group

Group A: Treatment

Experimental group

Description:

Vancomycin, magnesium citrate, microbiota

Treatment:

Combination Product: vancomycin, magnesium citrate, microbiota

Group B: Placebo

Placebo Comparator group

Description:

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

Treatment:

Combination Product: placebo vancomycin, real magnesium citrate, placebo microbiota