Microbiota Transfer Therapy for Pitt Hopkins Syndrome and GI Disorders

Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders: MTP-101 P

This study tests whether a new treatment using gut bacteria can help people with Pitt-Hopkins Syndrome who have gastrointestinal problems feel better.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 5 Years to 55 Years
• Sex: All
• Sponsor: Gut-Brain-Axis Therapeutics Inc. (Academic / other)
• Locations: 1 site (Tempe, Arizona)
• Trial ID: NCT06321796 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of Microbiota Transfer Therapy (MTT) in treating gastrointestinal issues in patients with Pitt-Hopkins Syndrome. The approach involves a combination of oral vancomycin, bowel cleansing, and a specific microbiota powder treatment over a 14-week period. Participants will be randomly assigned to receive either the treatment or a placebo in a double-blind manner, followed by an open-label observation phase. The study aims to evaluate the safety, tolerability, and efficacy of this novel therapy.

Who should consider this trial

Good fit: Ideal candidates are children aged 5-17 and adults aged 18-55 with confirmed Pitt-Hopkins Syndrome and chronic gastrointestinal disorders.

Not a fit: Patients who have not previously tried standard GI treatments or those with recent antibiotic use may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve gastrointestinal symptoms in patients with Pitt-Hopkins Syndrome.

How similar studies have performed: While the approach of microbiota transfer is gaining interest, this specific application for Pitt-Hopkins Syndrome is relatively novel and has not been extensively tested in prior studies.

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 (topical antibiotics are allowed)
2. Probiotics in 2 months prior to start of treatment, or fecal transplant in last 12 months. Foods naturally containing probiotics such as yogurt are allowed.
3. Tube feeding may be an exclusion criterion if the participant requires an unusual diet such as a liquid diet with low fiber.
4. Current severe gastrointestinal problems that require immediate hospital 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), or active malignancy or infection.
7. Recent or scheduled abdominal surgeries
8. Current participation in other clinical trials
9. Females who are pregnant or who are at risk of pregnancy and sexually active with a male partner without effective birth control. We will conduct a pregnancy test on all female participants 12 years and older as part of the screening and at each clinical visit.

   Males who are sexually active with a female partner without highly effective birth control (IUD or birth control hormones).
10. Allergy or intolerance to the study medications: vancomycin, magnesium citrate, milk powder with chocolate flavoring (which are included in MTP-101P), or the antacid.
11. Clinically significant abnormalities at baseline on the blood safety tests, and confirmed on a second test. The tests include Comprehensive Metabolic Panel, and Complete Blood Count with Differential. Note that some abnormalities may occur due to PTHS, so only those likely to significantly increase risk in this study would be grounds for exclusion, at the discretion of the study physician. See detailed discussion at the end of this section on Interpreting Laboratory Results. re. Eligibility for Admission to Study.
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.
13. Substantially decreased kidney function, as evidenced by estimated glomerular filtration rate of \<60 mL/min/1.73 m2. This is not normally reported for children on standard laboratory metabolic panels, so in those cases we will use the National Kidney Foundations Pediatric Glomerular filtration rate (GFR) Calculator to calculate the pediatric GFR based on age/height, the Blood urea nitrogen (BUN) and serum creatinine from our standard Comprehensive Metabolic Panel (CMP). \[https://www.kidney.org/professionals/kdoqi/gfr_calculatorped\] This calculator uses the Creatinine-based "Bedside Schwartz" equation (2009) that seems to be the most commonly used calculation for this purpose.
14. Participants who are breastfeeding.

Where this trial is running

Tempe, Arizona

• Autism/Asperger's Research Group at Arizona State University — Tempe, Arizona, United States (Recruiting)

Study contacts

• Study coordinator: James B Adams

Conditions Pitt Hopkins Syndrome; Microbiota Transfer Therapy; Fecal Transplant; Fecal Microbiota Transplant; Intestinal Microbiota