A Phase 2 interventional study of MTP-101P and Vancomycin in Pitt Hopkins Syndrome, sponsored by Gut-Brain-Axis Therapeutics Inc.. Recruiting at 1 site in United States. Open to participants aged 5 Years to 55 Years. Per ClinicalTrials.gov, last updated 2024-03-20.
Sponsored by Gut-Brain-Axis Therapeutics Inc. · Phase 2, Interventional, and Treatment
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.
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.
Gut-Brain-Axis Therapeutics Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Females who are pregnant or who are at risk of pregnancy and sexually active with a male partner without effective . 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).
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Part 1: Blinded Treatment (14 weeks) Vancomycin, Magnesium Citrate, Antacid, MTP-101P
Combination Product: MTP-101P · Combination Product: Vancomycin · Combination Product: Magnesium Citrate · Combination Product: Antacid
Part 1: Blinded Placebo (14 weeks) Placebo Vancomycin, Real Magnesium Citrate, Real Antacid, Placebo MTP-101P
Combination Product: Magnesium Citrate · Combination Product: Antacid · Combination Product: Placebo MTP-101P · Combination Product: Placebo Vancomycin
MTP-101P is comprised of standardized dose of total fecal microbiota purified from the stool of healthy donors. Donors are carefully screened via health status questionnaires, physical examinations, reviews of comprehensive medical history, clinical laboratory evaluations, serologic and genomic tests for infectious diseases and metabolic health, and stool-related pathogen testing. The material from the donors is purified to remove the majority of non-bacterial material, washed, lyophilized, and provided to the patient in a sachet contained within a mixing bottle. Duration: 12.5 Weeks
Oral vancomycin is an antibiotic used for treating C. difficile-associated diarrhea and staphylococcal-induced enterocolitis. Duration: 10 Days
Magnesium citrate is a common over-the-counter laxative and bowel cleanse. Duration: 1 Day
The antacid will be aluminum/magnesium hydroxide, 1x/day, 5 minutes before the MTP-101P. Each 5 ml consists of Aluminum Hydroxide - 200 mg, Magnesium Hydroxide - 200 mg, and Simethicone - 20 mg. If some participants cannot tolerate the aluminum/magnesium hydroxide, we will allow calcium carbonate alternatives equivalent in acid neutralizing capacity. Duration: 12.5 weeks
The same packaging will be used as for MTP-101P, but the sachets will contain a freeze-dried mixture of normal saline containing 10% Trehalose. Duration: 12.5 weeks
Same packaging and liquid carrier and flavoring used as for the oral vancomycin, but no vancomycin. Duration: 10 days
Daily Stool Record (DSR)
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.
Time frame: Baseline (for 14 days pre-treatment) vs End of Treatment (Days 82-95 of treatment).
Safety Measures (Adverse Events)
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.
Time frame: Days 0 to 99 of treatment
Gastrointestinal Symptom Rating Scale (GSRS)
An assessment of GI symptoms based on 15 questions, which are then scored in five domains (abdominal pain, reflux, indigestion, diarrhea, and constipation). Symptoms are rated on a 7-point Likert scale including no discomfort at all (1), slight discomfort (2), mild discomfort (3), moderate discomfort (4), moderately severe discomfort (5), severe discomfort (6), and very severe discomfort (7).
Time frame: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Clinical Global Impression - Gastrointestinal (CGI-GI)
This assessment is conducted by the study physician and assess the severity of GI symptoms on a 7-point scale. The severity scale rates the severity of the patient's illness including normal, not at all ill (1), borderline mentally ill (2), mildly ill (3), moderately ill (4), markedly ill (5), severely ill (6), among the most extremely ill patients (7).
Time frame: Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment)
Clinical Global Impression - Pitt Hopkins (CGI-PTHS)
This assessment is conducted by the study physician and assess the severity of Pitt Hopkins Symptoms on a 7-point scale. The severity scale rates the severity of the patient's illness including normal, not at all ill (1), borderline mentally ill (2), mildly ill (3), moderately ill (4), markedly ill (5), severely ill (6), among the most extremely ill patients (7).
Time frame: Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment).
Parent Global Impressions - Pitt Hopkins: Change in Symptoms (PGI-PTHS-2-Change)
An assessment of Pitt Hopkins Syndrome symptoms that rates the change in symptom severity since the start of treatment. Symptom severity is rated on a 7-point scale including much worse (-3), somewhat worse (-2), slightly worse (-1), no change (0), slightly better (1), somewhat better (2), and much better (3).
Time frame: End of Treatment (After 95 days of treatment)
Gastrointestinal Symptoms common in Pitt Hopkins (GI-PTHS)
Assess common symptoms in Pitt Hopkins, including 2 questions about toilet training, a question about GI feeding tubes, and 7 questions about current GI symptom severity including constipation, diarrhea, gas, bloating, pain, reflux/heartburn/regurgitation and vomiting. Symptoms are rated on a 7-point scale from none to extremely severe.
Time frame: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Revised Face Legs Activity Crying Consolability Pain Questionnaire (FLACC)
Modified from the original FLACC for children with cognitive impairment (Malviya et al., 2006). Assess pain in five areas (Face, Legs, Activity, Crying, Consolability) using a three-point scale for each area, resulting in a total FLACC pain scale ranging from zero to 10.
Time frame: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Benefit
Assess the effectiveness of MTT on overall benefit (no benefit (0)/slight benefit(1)/moderate benefit (2)/good benefit (3)/great benefit (4)).
Time frame: End of Treatment (After 95 days of treatment)
National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Adverse E
Assess the effectiveness of MTT on overall adverse effects of treatment (no adverse effects (0)/mild adverse effects (1)/moderate adverse effects (2)/severe adverse effects (3)).
Time frame: End of Treatment (After 95 days of treatment)
Plan to share: No
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Started Feb 2024 (actual). Primary completion is estimated for Feb 2025, 12 months after the start.
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Studies of the drug tested here: Fecal Microbiota Preparation MTP-101P
Pitt-Hopkins syndrome
Gut-Brain-Axis Therapeutics Inc.