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| Name | Class |
|---|---|
| Pitt Hopkins Research Foundation | OTHER |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group A: Treatment | Experimental | Vancomycin, magnesium citrate, microbiota |
|
| Group B: Placebo | Placebo Comparator | placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| vancomycin, magnesium citrate, microbiota | Combination Product | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 | weeks 0-14 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | change in score between baseline and week 14 |
| CGI for PTHS Symptoms |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arizona State University | Tempe | Arizona | 85284 | United States |
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| Label | URL |
|---|---|
| link for our research group | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Group A: Treatment | Vancomycin, magnesium citrate, microbiota 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 |
| FG001 | Group B: Placebo | placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota placebo vancomycin, real magnesium 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 |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Group A: Treatment | Vancomycin, magnesium citrate, microbiota 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 |
| BG001 | Group B: Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Mean | Standard Deviation | days | change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14) |
|
AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).
We use standard definition of the Adverse events and Serious Adverse events.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Group A: Treatment | Vancomycin, magnesium citrate, microbiota 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 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hyperactivity | Psychiatric disorders | MedDRA 12.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| James B. Adams | Arizona State University | 480 965-3316 | jim.adams@asu.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 28, 2022 | Feb 5, 2024 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| C537403 | Pitt-Hopkins syndrome |
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| ID | Term |
|---|---|
| D014640 | Vancomycin |
| C110422 | magnesium citrate |
| D064307 | Microbiota |
| ID | Term |
|---|---|
| D006020 | Glycopeptides |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D010455 | Peptides |
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Part 1: Randomized, double-blind, placebo-controlled Part 2: Treatment group enters observation, placebo group switched to treatment Part 3: Long-term observation
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Part 1 is double-blind Parts 2 and 3 are single-blind (outcomes assessor is blinded)
| placebo vancomycin, real magnesium citrate, placebo microbiota | Combination Product | 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 |
|
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. |
| 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). | 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. | 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). | change in score between baseline and week 14 |
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota placebo vancomycin, real magnesium 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 |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Daily Stool Record | Mean | Standard Deviation | Number of abnormal days out of 14 days |
|
placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota
placebo vancomycin, real magnesium 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
|
|
| Primary | Safety Measures | number of adverse events and serious adverse events likely associated with treatment | Posted | Number | number of AEs among all participants | weeks 0-14 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | change in score between baseline and week 14 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | change in score between baseline and week 14 |
|
|
|
| Secondary | 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). | Posted | Mean | Standard Deviation | -3 to +3 | change in score between baseline and week 14 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | change in score between baseline and week 14 |
|
|
|
| Secondary | 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). | Posted | Mean | Standard Deviation | units on a scale | change in score between baseline and week 14 |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| 3 |
| 3 |
| EG001 | Group B: Placebo | placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota placebo vancomycin, real magnesium 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 | 0 | 3 | 0 | 3 | 3 | 3 |
| Irritability | Psychiatric disorders | MedDRA 12.0 | Systematic Assessment |
|
| Gassy | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Insomnia | General disorders | MedDRA 12.0 | Systematic Assessment |
|
| Loss of appetite | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Redness- Genitalia | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
|
| Change in Stool Color | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Abdominal Pain | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Bloating | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
|
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| D000602 |
| Amino Acids, Peptides, and Proteins |
| D008827 | Microbiological Phenomena |
| D058448 | Biota |
| D044822 | Biodiversity |
| D017753 | Ecosystem |
| D004777 | Environment |
| D055669 | Ecological and Environmental Phenomena |
| D001686 | Biological Phenomena |
| D004778 | Environment and Public Health |