MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Pitt Hopkins Syndrome Clinical Trial

Official title:

Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04132427
• Other study ID #: IRB#: 120190263
• Status: Completed
• Phase: Phase 2
• Start date: September 30, 2019
• Est. completion date: April 15, 2022

Study information

• Verified date: January 2023
• Source: Arizona State University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Description:

For children ages 5-17 years with PTHS and gastrointestinal problems, a a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT. The three parts of this trial are described below.

Part 1: Placebo-Controlled Treatment (14 weeks) The trial will begin with 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

Part 2 Open-Label Observation and Cross-Over (14 weeks) Group 1: Observation over the next 14 weeks (no additional treatment) Group 2: They will receive the same treatment that group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, an initial high dose of FM for 4 days, and then a lower dose of FM for 12 weeks.

Part 3: Follow-up There will be a follow-up evaluation at 14 weeks after the end of part 2, to assess long-term efficacy and possible adverse effects.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: April 15, 2022
• Est. primary completion date: March 17, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Digestive System Diseases
• Facies
• Gastrointestinal Diseases
• Hyperventilation
• Intellectual Disability
• Pitt Hopkins Syndrome
• Syndrome

Intervention

Combination Product:
• 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
• placebo vancomycin, real magnsium 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

Locations

Country	Name	City	State
United States	Arizona State University	Tempe	Arizona

Sponsors (2)

Lead Sponsor	Collaborator
Arizona State University	Pitt Hopkins Research Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Daily Stool Record (DSR(	The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the % of days with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms..	change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)
Primary	Safety Measures	number of adverse events and serious adverse events likely associated with treatment	weeks 0-14
Secondary	CGI for GI Disorders	Clinical Global Impressions of GI Disorders, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.	change in score between baseline and week 14
Secondary	CGI for PTHS Symptoms	Clinical Global Impressions of Pitt Hopkins Syndrome symptoms, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.	change in score between baseline and week 14
Secondary	PGI-PTHS	Parent Global Impressions of Pitt Hopkins Syndrome symptoms. This is a rating of 29 symptoms, and an average is computed. The scale ranges from -3 (much worse) to +3 (much better).	change in score between baseline and week 14
Secondary	GSRS	Gastrointestinal Symptom Rating Scale. This is a 15-item questionnaire, with each item rated on a scale of 1 (no symptoms) to 7 (very severe discomfort).	change in score between baseline and week 14
Secondary	FLACC	Revised Face Legs Activity Crying Consolability Pain Questionnaire for Children with Cognitive Impairment (FLACC). This is a rating scale of 5 symptoms of pain, with each item rated on a scale of 0 (no symptom) to 2 (maximum symptom), and the scores for each item are summed to create a total score.	change in score between baseline and week 14

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