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Recruiting Phase 2 NCT06321796

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

Trial Parameters

Condition Pitt Hopkins Syndrome
Sponsor Gut-Brain-Axis Therapeutics Inc.
Study Type INTERVENTIONAL
Phase Phase 2
Enrollment 20
Sex ALL
Min Age 5 Years
Max Age 55 Years
Start Date 2024-02-27
Completion 2025-02
Interventions
MTP-101PVancomycinMagnesium Citrate

Brief Summary

The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt-Hopkins Syndrome (PTHS) and gastrointestinal problems (constipation, bloating, abdominal pain). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using magnesium citrate, followed by 4 days of high dose MTP-101P with an antacid, followed by 12 weeks of a lower maintenance dose of MTP-101P with an antacid.

Eligibility Criteria

Inclusion Criteria: 1. Children ages 5 to \<18 years and adults ages 18 to \< 55 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 the 2 months prior to start of treatment, and no intention to change treatments during Part 1 (all participants) and Part 2 (group B) of the clinical trial. The only exception is GI medications, which may be reduced if symptoms reduce, and any changes during the study will be documented. 4. Review of last year of medical records by the study physician. 5. At least two previous trials of "standard of care" GI treatments that did not alleviate GI symptoms (constipation, diarrhea, bloating, gas, reflux, and/or abdominal pain). Standard of care treatments include laxatives, stool softeners, enemas, suppositories, or similar medications. Exclusion Criteria: 1. Antibiotics in 2 months prior to start of treatment (topi

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