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Trial record 1 of 1 for:    NCT04132427
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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
Sponsor:
Collaborator:
Pitt Hopkins Research Foundation
Information provided by (Responsible Party):
Arizona State University

Brief 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). 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).

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

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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..

  2. Safety Measures [ Time Frame: weeks 0-14 ]
    number of adverse events and serious adverse events likely associated with treatment


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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).

  4. 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).

  5. 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.



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Ages Eligible for Study:   7 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)
  2. GI disorder as defined below that has lasted for at least 2 years.
  3. 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.
  4. Ability to swallow pills (without chewing)
  5. Review of last two years of medical records by the study physician.

Exclusion Criteria:

  1. Antibiotics in last 3 months
  2. Probiotics in last 2 months, or fecal transplant in last 12 months
  3. Tube feeding
  4. Severe gastrointestinal problems that require immediate treatment (life-threatening)
  5. Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions
  6. Unstable, poor health (based on study physician's opinion)
  7. Recent or scheduled surgeries
  8. Current participation in other clinical trials
  9. Females who are pregnant or who are at risk of pregnancy and sexually active without effective birth control.
  10. Allergy or intolerance to vancomycin or magnesium citrate
  11. Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel, and Complete Blood Count with Differential.
  12. 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.

Information from the National Library of Medicine

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


Contacts
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Contact: James B Adams, PhD 4809653316 jim.adams@asu.edu
Contact: Chelsie Caruso, BS 8314020283 cmcarus3@asu.edu

Locations
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United States, Arizona
Arizona State University Recruiting
Tempe, Arizona, United States, 85284
Contact: James B Adams, PhD    480-965-3316    jim.adams@asu.edu   
Contact: Chelsie Caruso, BS    8314020283    cmcarus3@asu.edu   
Sponsors and Collaborators
Arizona State University
Pitt Hopkins Research Foundation
Additional Information:
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Responsible Party: Arizona State University
ClinicalTrials.gov Identifier: NCT04132427    
Other Study ID Numbers: IRB#: 120190263
First Posted: October 18, 2019    Key Record Dates
Last Update Posted: October 30, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Arizona State University:
microbiota transfer therapy
fecal transplant
fecal microbiota transplant
intestinal microbiota
Additional relevant MeSH terms:
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Digestive System Diseases
Gastrointestinal Diseases
Hyperventilation
Intellectual Disability
Syndrome
Facies
Disease
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
Signs and Symptoms, Respiratory
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Neurodevelopmental Disorders
Mental Disorders
Disease Attributes
Vancomycin
Magnesium citrate
Citric Acid
Sodium Citrate
Anticoagulants
Calcium Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Anti-Bacterial Agents
Anti-Infective Agents
Cathartics
Gastrointestinal Agents