Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders: MTP-101 P
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Pitt Hopkins Syndrome
- Sponsor
- Gut-Brain-Axis Therapeutics Inc.
- Enrollment
- 20
- Locations
- 1
- Primary Endpoint
- Safety Measures (Adverse Events)
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
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.
Detailed Description
For children 5-17 years old and adults 18-55 years old with Pitt-Hopkins Syndrome (PTHS) and Gastrointestinal (GI) problems who have attempted two standard-of-care (SOC) GI treatments with no alleviation of symptoms. This is a Phase 2 clinical trial that will evaluate the safety, tolerability, and efficacy of a powder version of Microbiota Transfer Therapy (MTT) called MTP-101P. 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 4 days of initial high dose of MTP-101P taken daily 5 minutes after antacid, and then 12 weeks of a lower maintenance dose of MTP-101P taken daily 5 minutes after an antacid. Group A: Real Treatment vs. Group B: Placebo vancomycin, real magnesium citrate, placebo MTP-101P, real antacid. Part 2: Open-Label Observation and Cross-Over (14 weeks) Group A: Observation over the next 14 weeks (no additional treatment). Group A completes the study at the end of part 2. Group B: They will receive the same treatment that Group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, and an initial high dose of MTP-101P for 4 days taken daily 5 minutes after an antacid, and then a lower dose of MTP-101P for 12 weeks taken daily 5 minutes after antacid. Part 3: Follow Up Group B: There will be a follow-up evaluation 14 weeks post-treatment after the end of Part 2, to assess long-term efficacy and possible adverse effects.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Children ages 5 to \<18 years and adults ages 18 to \< 55 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 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.
- •Review of last year of medical records by the study physician.
- •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
- •Antibiotics in 2 months prior to start of treatment (topical antibiotics are allowed)
- •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.
- •Tube feeding may be an exclusion criterion if the participant requires an unusual diet such as a liquid diet with low fiber.
- •Current severe gastrointestinal problems that require immediate hospital 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), or active malignancy or infection.
- •Recent or scheduled abdominal surgeries
- •Current participation in other clinical trials
- •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).
Outcomes
Primary Outcomes
Safety Measures (Adverse Events)
Time Frame: Days 0 to 99 of treatment
Safety will be assessed based on the number of adverse events during treatment for each group (Group A and Group B). Units are number of adverse events.
Daily Stool Record (DSR)
Time Frame: Baseline (for 14 days pre-treatment) vs End of Treatment (Days 82-95 of treatment).
Participants will report the number of abnormal events during a 14 day period. An event includes no bowel movement during 1 day, unusually hard stool (Bristol Stool Form type 1-2), unusually soft stool (Bristol Stool Form type 6-7), four or more bowel movements in 1 day, abdominal pain, or use of GI medication. The units are number of events regardless of type of event.
Secondary Outcomes
- Clinical Global Impression - Gastrointestinal (CGI-GI)(Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment))
- Parent Global Impressions - Pitt Hopkins: Change in Symptoms (PGI-PTHS-2-Change)(End of Treatment (After 95 days of treatment))
- Clinical Global Impression - Pitt Hopkins (CGI-PTHS)(Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment).)
- Revised Face Legs Activity Crying Consolability Pain Questionnaire (FLACC)(Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment))
- Gastrointestinal Symptom Rating Scale (GSRS)(Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment))
- National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Adverse E(End of Treatment (After 95 days of treatment))
- Gastrointestinal Symptoms common in Pitt Hopkins (GI-PTHS)(Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment))
- National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Benefit(End of Treatment (After 95 days of treatment))