Trial · NCT04132427
MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
NCT04132427, Phase 2, Completed, Arizona State University; results first posted 2024-08-26
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders. Trials, record NCT04132427. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04132427
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
- NCT number
- NCT04132427
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- Arizona State University
- Conditions
- Pitt Hopkins Syndrome
- Interventions
- vancomycin, magnesium citrate, microbiota, placebo vancomycin, real magnesium citrate, placebo microbiota
- First posted
- 2019-10-18
- Last updated
- 2024-08-26
- Results first posted
- 2024-08-26
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Daily Stool Record (DSR( | 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 number of days (out of 14 days) with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms. |
| Primary | Safety Measures | weeks 0-14 | number of adverse events and serious adverse events likely associated with treatment |
| Secondary | CGI for GI Disorders | 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. |
| Secondary | CGI for PTHS Symptoms | 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. |
| Secondary | PGI-PTHS | 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). |
| Secondary | GSRS | 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). The average score for all 15 items is reported here. |
| Secondary | FLACC | 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 (zero to 10). |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Daily Stool Record (DSR( | change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14) | days | MEAN | |
| PRIMARY | Safety Measures | weeks 0-14 | number of AEs among all participants | NUMBER | |
| SECONDARY | CGI for GI Disorders | change in score between baseline and week 14 | units on a scale | MEAN | |
| SECONDARY | CGI for PTHS Symptoms | change in score between baseline and week 14 | units on a scale | MEAN | |
| SECONDARY | PGI-PTHS | change in score between baseline and week 14 | -3 to +3 | MEAN | |
| SECONDARY | GSRS | change in score between baseline and week 14 | units on a scale | MEAN | |
| SECONDARY | FLACC | change in score between baseline and week 14 | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Daily Stool Record (DSR( | Group A: Treatment | -2.3 | Standard Deviation 2.5 | days | |
| Daily Stool Record (DSR( | Group B: Placebo | 0 | Standard Deviation 0 | days | |
| Safety Measures | Group A: Treatment | 8 | number of AEs among all participants | ||
| Safety Measures | Group B: Placebo | 10 | number of AEs among all participants | ||
| CGI for GI Disorders | Group A: Treatment | -1 | Standard Deviation 1 | units on a scale | |
| CGI for GI Disorders | Group B: Placebo | -.67 | Standard Deviation 1.15 | units on a scale | |
| CGI for PTHS Symptoms | Group A: Treatment | 0 | Standard Deviation 0 | units on a scale | |
| CGI for PTHS Symptoms | Group B: Placebo | -.33 | Standard Deviation .58 | units on a scale | |
| PGI-PTHS | Group A: Treatment | .68 | Standard Deviation .97 | -3 to +3 | |
| PGI-PTHS | Group B: Placebo | .29 | Standard Deviation .44 | -3 to +3 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Group A: Treatment | 3 | |
| Overall Study | STARTED | Group B: Placebo | 3 | |
| Overall Study | COMPLETED | Group A: Treatment | 3 | |
| Overall Study | COMPLETED | Group B: Placebo | 3 | |
| Overall Study | NOT COMPLETED | Group A: Treatment | 0 | |
| Overall Study | NOT COMPLETED | Group B: Placebo | 0 |
Source ClinicalTrials.gov
Adverse events22
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hyperactivity | Psychiatric disorders | Group A: Treatment | 1 | 3 | 1 |
| other | Hyperactivity | Psychiatric disorders | Group B: Placebo | 2 | 3 | 2 |
| other | Irritability | Psychiatric disorders | Group A: Treatment | 1 | 3 | 1 |
| other | Irritability | Psychiatric disorders | Group B: Placebo | 2 | 3 | 2 |
| other | Gassy | Gastrointestinal disorders | Group A: Treatment | 2 | 3 | 2 |
| other | Gassy | Gastrointestinal disorders | Group B: Placebo | 2 | 3 | 2 |
| other | Insomnia | General disorders | Group A: Treatment | 1 | 3 | 1 |
| other | Insomnia | General disorders | Group B: Placebo | 2 | 3 | 2 |
| other | Loss of appetite | Gastrointestinal disorders | Group A: Treatment | 1 | 3 | 1 |
| other | Loss of appetite | Gastrointestinal disorders | Group B: Placebo | 1 | 3 | 1 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Group A: Treatment | 0 | 3 |
| Total, all-cause mortality | Group B: Placebo | 0 | 3 |
| Total, other adverse events | Group A: Treatment | 3 | 3 |
| Total, other adverse events | Group B: Placebo | 3 | 3 |
| Total, serious adverse events | Group A: Treatment | 0 | 3 |
| Total, serious adverse events | Group B: Placebo | 0 | 3 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Group A: Treatment vs Group B: Placebo: deaths from any cause | Trial comparisons |
| Group A: Treatment vs Group B: Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04132427
GET https://conductscience.org/api/trials/v1/NCT04132427/events?page=2