A Phase 2 interventional study of vancomycin, magnesium citrate, microbiota and placebo vancomycin, real magnesium citrate, placebo microbiota in Pitt Hopkins Syndrome, sponsored by Arizona State University. Completed at 1 site in United States. Open to participants aged 7 Years to 17 Years. Per ClinicalTrials.gov, last updated 2024-08-26.
Sponsored by Arizona State University · 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 similar to Irritable Bowel Syndrome (IBS). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by a bowel cleanse, followed by 12 weeks of Fecal Microbiota (FM).
For children ages 5-17 years with PTHS and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT.
Randomized, double-blind, placebo-Controlled Treatment (14 weeks)
The trial will be 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
Arizona State University is the lead sponsor of 238 studies on the registry; 42 are 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:
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.
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.
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.
Vancomycin, magnesium citrate, microbiota
Combination Product: vancomycin, magnesium citrate, microbiota
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
Combination Product: placebo vancomycin, real magnesium citrate, placebo 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
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(
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.
Time frame: change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)
Safety Measures
number of adverse events and serious adverse events likely associated with treatment
Time frame: weeks 0-14
CGI for GI Disorders
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.
Time frame: change in score between baseline and week 14
CGI for PTHS Symptoms
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.
Time frame: change in score between baseline and week 14
PGI-PTHS
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).
Time frame: change in score between baseline and week 14
GSRS
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.
Time frame: change in score between baseline and week 14
FLACC
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).
Time frame: change in score between baseline and week 14
| Milestone | Group A: Treatment | Group B: Placebo |
|---|---|---|
| Started | 3 | 3 |
| Completed | 3 | 3 |
| Not completed | 0 | 0 |
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.
| days | Group A: Treatment | Group B: Placebo |
|---|---|---|
| Daily Stool Record (DSR( | -2.3 ± 2.5 | 0 ± 0 |
number of adverse events and serious adverse events likely associated with treatment
| number of AEs among all participants | Group A: Treatment | Group B: Placebo |
|---|---|---|
| Safety Measures | 8 | 10 |
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.
| units on a scale | Group A: Treatment | Group B: Placebo |
|---|---|---|
| CGI for GI Disorders | -1 ± 1 | -.67 ± 1.15 |
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.
| units on a scale | Group A: Treatment | Group B: Placebo |
|---|---|---|
| CGI for PTHS Symptoms | 0 ± 0 | -.33 ± .58 |
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).
| -3 to +3 | Group A: Treatment | Group B: Placebo |
|---|---|---|
| PGI-PTHS | .68 ± .97 | .29 ± .44 |
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.
| units on a scale | Group A: Treatment | Group B: Placebo |
|---|---|---|
| GSRS | -2.3 ± .87 | -1.07 ± 1.91 |
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).
| units on a scale | Group A: Treatment | Group B: Placebo |
|---|---|---|
| FLACC | -4.0 ± 2.65 | -2.67 ± 2.08 |
Collected over AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Group A: Treatment | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Group B: Placebo | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Event | Group A: Treatment | Group B: Placebo |
|---|---|---|
| Abdominal PainGastrointestinal disorders | 1/3 | 3/3 |
| BloatingGastrointestinal disorders | 0/3 | 3/3 |
| ConstipationGastrointestinal disorders | 1/3 | 3/3 |
| HyperactivityPsychiatric disorders | 1/3 | 2/3 |
| IrritabilityPsychiatric disorders | 1/3 | 2/3 |
| GassyGastrointestinal disorders | 2/3 | 2/3 |
| InsomniaGeneral disorders | 1/3 | 2/3 |
| DiarrheaGastrointestinal disorders | 2/3 | 2/3 |
| Change in Stool ColorGastrointestinal disorders | 2/3 | 1/3 |
| Loss of appetiteGastrointestinal disorders | 1/3 | 1/3 |
| Age, Categorical(Participants) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| <=18 years | 3 | 3 | 6 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Age, Continuous(years) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| Mean | 13.0 ± 3.6 | 12.7 ± 3.1 | 12.8 ± 3.0 |
| Sex: Female, Male(Participants) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| Female | 1 | 1 | 2 |
| Male | 2 | 2 | 4 |
| Ethnicity (NIH/OMB)(Participants) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 1 |
| Not Hispanic or Latino | 2 | 3 | 5 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 3 | 3 | 6 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| United States | 3 | 3 | 6 |
| Daily Stool Record(Number of abnormal days out of 14 days) | Group A: Treatment | Group B: Placebo | Total |
|---|---|---|---|
| Mean | 14 ± 0 | 14 ± 0 | 14 ± 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Pitt-Hopkins syndrome