Pitt Hopkins SyndromePhase 2NCT04132427
MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
A Phase 2 study testing vancomycin, magnesium citrate, microbiota in people with Pitt Hopkins Syndrome, run by Arizona State University at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 7 to 17 years
- Conditions the study is for
- Pitt Hopkins Syndrome
- What is being tested?
- vancomycin, magnesium citrate, microbiota
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2019to 15 Apr 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)
- GI disorder as defined below that has lasted for at least 2 years.
- No changes in medications, supplements, diet, or therapies in last 2 months, and no intention to change them during the Parts 1 and 2 of the clinical trial.
- Ability to swallow pills (without chewing)
- Review of last two years of medical records by the study physician.
Who cannot join
- Antibiotics in last 3 months
- Probiotics in last 2 months, or fecal transplant in last 12 months
- Tube feeding
- Severe gastrointestinal problems that require immediate treatment (life-threatening)
- Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
- Unstable, poor health (based on study physician's opinion)
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- Recent or scheduled surgeries
- Current participation in other clinical trials
- Females who are pregnant or who are at risk of pregnancy and sexually active without effective birth control.
- Allergy or intolerance to vancomycin or magnesium citrate
- Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel, and Complete Blood Count with Differential.
- Evidence of significant impairment of immune system, or taking medications that can compromise the immune system, and thus increase risk if exposed to multiple-drug resistant bacteria.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
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).
Read the detailed description
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
- Conditions
- Pitt Hopkins Syndrome
- Being tested
- vancomycin, magnesium citrate, microbiota, placebo vancomycin, real magnesium citrate, placebo microbiota
- Keywords
- microbiota transfer therapy, fecal transplant, fecal microbiota transplant, intestinal microbiota
- Medical subject headings
- Pitt-Hopkins syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vancomycin, magnesium citrate, microbiotaCombination 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
placebo vancomycin, real magnesium citrate, placebo microbiotaCombination 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
| Study group | Role | What they receive |
|---|---|---|
| Group A: Treatment | Experimental | Vancomycin, magnesium citrate, microbiota Receives: Combination Product: vancomycin, magnesium citrate, microbiota |
| Group B: Placebo | Placebo comparator | placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota Receives: Combination Product: placebo vancomycin, real magnesium citrate, placebo microbiota |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: weeks 0-14
Secondary outcomes (5)
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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: change in score between baseline and week 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2019
- Study start30 Sep 2019
- Primary completion17 Mar 2022
- Study completion15 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pitt Hopkins SyndromeStudies of the same condition, open and under way.
- More trials run by Arizona State UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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 blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arizona State University
- Sponsor type
- University, hospital or other
- Collaborators
- Pitt Hopkins Research Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04132427
- Sponsor's protocol ID
- IRB#: 120190263
- Status last verified
- Mar 2024
- First submitted
- 11 Oct 2019
- Last updated
- 26 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
- ClinicalTrials.gov ID
- NCT04132427
- Acronym / sponsor ID
- IRB#: 120190263
- Conditions
- Pitt Hopkins Syndrome
- Keywords
- microbiota transfer therapy, fecal transplant, fecal microbiota transplant, intestinal microbiota
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Arizona State UniversityUniversity, hospital or other
- Collaborators
- Pitt Hopkins Research Foundation
- Study start
- 30 Sep 2019 (actual)
- Primary completion
- 17 Mar 2022 (actual)
- Study completion
- 15 Apr 2022 (actual)
- First posted
- 18 Oct 2019 (actual)
- Results
- Posted 26 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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
| Arm | Type | Receives |
|---|---|---|
| Group A: Treatment | Experimental | Combination Product: vancomycin, magnesium citrate, microbiotaVancomycin, magnesium citrate, microbiota |
| Group B: Placebo | Placebo comparator | Combination Product: placebo vancomycin, real magnesium citrate, placebo microbiotaplacebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota |
Who may qualify
- Ages
- 7 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)
- GI disorder as defined below that has lasted for at least 2 years.
- No changes in medications, supplements, diet, or therapies in last 2 months, and no intention to change them during the Parts 1 and 2 of the clinical trial.
- Ability to swallow pills (without chewing)
- Review of last two years of medical records by the study physician.
Key exclusion criteria (12 listed)
- Antibiotics in last 3 months
- Probiotics in last 2 months, or fecal transplant in last 12 months
- Tube feeding
- Severe gastrointestinal problems that require immediate treatment (life-threatening)
- Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vancomycin, magnesium citrate, microbiota10 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 | Combination product | Group A: Treatment |
| placebo vancomycin, real magnesium citrate, placebo microbiota10 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 | Combination product | Group B: Placebo |
Primary outcome
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 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.
Safety Measures
Time frame: weeks 0-14
number of adverse events and serious adverse events likely associated with treatment
5 secondary outcomes
CGI for GI Disorders
Time frame: change in score between baseline and week 14
CGI for PTHS Symptoms
Time frame: change in score between baseline and week 14
PGI-PTHS
Time frame: change in score between baseline and week 14
GSRS
Time frame: change in score between baseline and week 14
FLACC
Time frame: change in score between baseline and week 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- link for our research groupLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing vancomycin, magnesium citrate, microbiota in people with Pitt Hopkins Syndrome, run by Arizona State University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2024.