MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

NCT04132427 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: IRB#: 120190263
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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).

Conditions

Pitt Hopkins Syndrome

Keywords

microbiota transfer therapy; fecal transplant; fecal microbiota transplant; intestinal microbiota

Outcome Measures

Primary Outcomes (2)

• Daily Stool Record (DSR(

  The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the number of days (out of 14 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)

• Safety Measures

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

  weeks 0-14

Secondary Outcomes (5)

• CGI for GI Disorders

  change in score between baseline and week 14

• CGI for PTHS Symptoms

  change in score between baseline and week 14

• PGI-PTHS

  change in score between baseline and week 14

• GSRS

  change in score between baseline and week 14

• FLACC

  change in score between baseline and week 14

Study Arms (2)

Group A: Treatment

EXPERIMENTAL

Vancomycin, magnesium citrate, microbiota

Combination Product: vancomycin, magnesium citrate, microbiota

Group B: Placebo

PLACEBO COMPARATOR

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

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

Interventions

vancomycin, magnesium citrate, microbiota 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 A: Treatment

placebo vancomycin, real magnesium citrate, placebo microbiota 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

Group B: Placebo

Eligibility Criteria

• Age: 7 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Arizona State University: lead
• Pitt Hopkins Research Foundation: collaborator

Study Sites (1)

Arizona State University

Tempe, Arizona, 85284, United States

Location

Related Links

• link for our research group

MeSH Terms

Conditions

Pitt-Hopkins syndrome

Interventions

Vancomycin; magnesium citrate; Microbiota

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins; Microbiological Phenomena; Biota; Biodiversity; Ecosystem; Environment; Ecological and Environmental Phenomena; Biological Phenomena; Environment and Public Health

Results Point of Contact

• Title: James B. Adams
• Organization: Arizona State University

jim.adams@asu.edu

480 965-3316

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Part 1 is double-blind Parts 2 and 3 are single-blind (outcomes assessor is blinded)
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Part 1: Randomized, double-blind, placebo-controlled Part 2: Treatment group enters observation, placebo group switched to treatment Part 3: Long-term observation

Study Record Dates

• First Submitted: October 11, 2019
• First Posted: October 18, 2019
• Study Start: September 30, 2019
• Primary Completion: March 17, 2022
• Study Completion: April 15, 2022
• Last Updated: August 26, 2024
• Results First Posted: August 26, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)