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MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Primary Purpose

Pitt Hopkins Syndrome

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

vancomycin, magnesium citrate, microbiota

placebo vancomycin, real magnsium citrate, placebo microbiota

About this trial

This is an interventional treatment trial for Pitt Hopkins Syndrome focused on measuring microbiota transfer therapy, fecal transplant, fecal microbiota transplant, intestinal microbiota

Eligibility Criteria

7 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Arizona State University

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Group A: Treatment

Group B: Placebo

Arm Description

Vancomycin, magnesium citrate, microbiota

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

Outcomes

Primary Outcome Measures

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..

Safety Measures

number of adverse events and serious adverse events likely associated with treatment

Secondary Outcome Measures

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.

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.

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).

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).

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.

Full Information

NCT ID

NCT04132427

First Posted

October 11, 2019

Last Updated

January 6, 2023

Sponsor

Arizona State University

Collaborators

Pitt Hopkins Research Foundation

1. Study Identification

Unique Protocol Identification Number

NCT04132427

Brief Title

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

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Completed

Study Start Date

September 30, 2019 (Actual)

Primary Completion Date

March 17, 2022 (Actual)

Study Completion Date

April 15, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Arizona State University

Collaborators

Pitt Hopkins Research Foundation

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pitt Hopkins Syndrome

Keywords

microbiota transfer therapy, fecal transplant, fecal microbiota transplant, intestinal microbiota

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

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

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Masking Description

Part 1 is double-blind Parts 2 and 3 are single-blind (outcomes assessor is blinded)

Allocation

Randomized

Enrollment

7 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Group A: Treatment

Arm Type

Experimental

Arm Description

Vancomycin, magnesium citrate, microbiota

Arm Title

Group B: Placebo

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Combination Product

Intervention Name(s)

vancomycin, magnesium citrate, microbiota

Intervention Description

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

Intervention Name(s)

placebo vancomycin, real magnsium citrate, placebo microbiota

Intervention Description

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

Primary Outcome Measure Information:

Title

Daily Stool Record (DSR(

Description

Time Frame

change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)

Title

Safety Measures

Description

number of adverse events and serious adverse events likely associated with treatment

Time Frame

weeks 0-14

Secondary Outcome Measure Information:

Title

CGI for GI Disorders

Description

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.

Time Frame