MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

NCT ID: NCT04132427

Last Updated: 2024-08-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-30
• Study Completion Date: 2022-04-15

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

Conditions

Pitt Hopkins Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Group A: Treatment

Vancomycin, magnesium citrate, microbiota

Group Type: EXPERIMENTAL

vancomycin, magnesium citrate, microbiota

Intervention Type: COMBINATION_PRODUCT

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

Group B: Placebo

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

Group Type: PLACEBO_COMPARATOR

placebo vancomycin, real magnesium citrate, placebo microbiota

Intervention Type: COMBINATION_PRODUCT

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

Interventions

vancomycin, magnesium citrate, microbiota

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

Intervention Type: COMBINATION_PRODUCT

placebo vancomycin, real magnesium 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

Intervention Type: COMBINATION_PRODUCT

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.

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pitt Hopkins Research Foundation

OTHER

Sponsor Role: collaborator

Arizona State University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Arizona State University

Tempe, Arizona, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

https://autism.asu.edu

link for our research group

Other Identifiers

IRB#: 120190263

Identifier Type: -

Identifier Source: org_study_id