MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
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|ClinicalTrials.gov Identifier: NCT04132427|
Recruitment Status : Recruiting
First Posted : October 18, 2019
Last Update Posted : October 30, 2020
|Condition or disease||Intervention/treatment||Phase|
|Pitt Hopkins Syndrome||Combination Product: vancomycin, magnesium citrate, microbiota Combination Product: placebo vancomycin, real magnsium citrate, placebo microbiota||Phase 2|
For children ages 5-17 years with PTHS and gastrointestinal problems, a a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT. 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 oral administration of FM (or placebo). An initial high dose of FM (or placebo) for two days will be followed by a lower maintenance dose of FM (or placebo) for 12 weeks.
Group A: real treatment Group B: placebo vancomycin, real magnesium citrate, placebo FM
Part 2 Open-Label Observation and Cross-Over (14 weeks) Group 1: Observation over the next 14 weeks (no additional treatment) Group 2: They will receive the same treatment that group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, an initial high dose of FM for 4 days, and then a lower dose of FM for 12 weeks.
Part 3: Follow-up There will be a follow-up evaluation at 14 weeks after the end of part 2, to assess long-term efficacy and possible adverse effects.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Part 1: Randomized, double-blind, placebo-controlled Part 2: Treatment group enters observation, placebo group switched to treatment Part 3: Long-term observation|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||Part 1 is double-blind Parts 2 and 3 are single-blind (outcomes assessor is blinded)|
|Official Title:||Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders|
|Actual Study Start Date :||September 30, 2019|
|Estimated Primary Completion Date :||February 10, 2022|
|Estimated Study Completion Date :||February 10, 2022|
Experimental: Group A: Treatment
Vancomycin, magnesium citrate, microbiota
Combination Product: vancomycin, magnesium citrate, microbiota
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
Placebo Comparator: Group B: Placebo
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
Combination Product: placebo vancomycin, real magnsium citrate, placebo microbiota
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
- Daily Stool Record (DSR( [ Time Frame: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14) ]The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the % of days with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms..
- Safety Measures [ Time Frame: weeks 0-14 ]number of adverse events and serious adverse events likely associated with treatment
- CGI for GI Disorders [ Time Frame: change in score between baseline and week 14 ]Clinical Global Impressions of GI Disorders, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.
- CGI for PTHS Symptoms [ Time Frame: change in score between baseline and week 14 ]Clinical Global Impressions of Pitt Hopkins Syndrome symptoms, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.
- PGI-PTHS [ Time Frame: change in score between baseline and week 14 ]Parent Global Impressions of Pitt Hopkins Syndrome symptoms. This is a rating of 29 symptoms, and an average is computed. The scale ranges from -3 (much worse) to +3 (much better).
- GSRS [ Time Frame: change in score between baseline and week 14 ]Gastrointestinal Symptom Rating Scale. This is a 15-item questionnaire, with each item rated on a scale of 1 (no symptoms) to 7 (very severe discomfort).
- FLACC [ Time Frame: change in score between baseline and week 14 ]Revised Face Legs Activity Crying Consolability Pain Questionnaire for Children with Cognitive Impairment (FLACC). This is a rating scale of 5 symptoms of pain, with each item rated on a scale of 0 (no symptom) to 2 (maximum symptom), and the scores for each item are summed to create a total score.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04132427
|Contact: James B Adams, PhDemail@example.com|
|Contact: Chelsie Caruso, BSfirstname.lastname@example.org|