Trials Sponsors Conditions Drugs Pricing

Conditions

Pitt Hopkins Syndrome

Keywords

Microbiota Transfer TherapyFecal TransplantFecal Microbiota TransplantIntestinal Microbiota

Outcome Measures

Primary Outcomes (2)

Daily Stool Record (DSR)
Participants will report the number of abnormal events during a 14 day period. An event includes no bowel movement during 1 day, unusually hard stool (Bristol Stool Form type 1-2), unusually soft stool (Bristol Stool Form type 6-7), four or more bowel movements in 1 day, abdominal pain, or use of GI medication. The units are number of events regardless of type of event.
Baseline (for 14 days pre-treatment) vs End of Treatment (Days 82-95 of treatment).
Safety Measures (Adverse Events)
Safety will be assessed based on the number of adverse events during treatment for each group (Group A and Group B). Units are number of adverse events.
Days 0 to 99 of treatment

Secondary Outcomes (8)

Gastrointestinal Symptom Rating Scale (GSRS)
Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Clinical Global Impression - Gastrointestinal (CGI-GI)
Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment)
Clinical Global Impression - Pitt Hopkins (CGI-PTHS)
Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment).
Parent Global Impressions - Pitt Hopkins: Change in Symptoms (PGI-PTHS-2-Change)
End of Treatment (After 95 days of treatment)
Gastrointestinal Symptoms common in Pitt Hopkins (GI-PTHS)
Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
+3 more secondary outcomes

Study Arms (2)

Group A: Treatment

EXPERIMENTAL

Part 1: Blinded Treatment (14 weeks) Vancomycin, Magnesium Citrate, Antacid, MTP-101P

Combination Product: MTP-101PCombination Product: VancomycinCombination Product: Magnesium CitrateCombination Product: Antacid

Group B: Placebo

PLACEBO COMPARATOR

Part 1: Blinded Placebo (14 weeks) Placebo Vancomycin, Real Magnesium Citrate, Real Antacid, Placebo MTP-101P

Combination Product: Magnesium CitrateCombination Product: AntacidCombination Product: Placebo MTP-101P

Interventions

MTP-101PCombination product

MTP-101P is comprised of standardized dose of total fecal microbiota purified from the stool of healthy donors. Donors are carefully screened via health status questionnaires, physical examinations, reviews of comprehensive medical history, clinical laboratory evaluations, serologic and genomic tests for infectious diseases and metabolic health, and stool-related pathogen testing. The material from the donors is purified to remove the majority of non-bacterial material, washed, lyophilized, and provided to the patient in a sachet contained within a mixing bottle. Duration: 12.5 Weeks

Group A: Treatment

VancomycinCombination product

Eligibility Criteria

Age5 Years - 55 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

Children ages 5 to \<18 years and adults ages 18 to \< 55 years with Pitt Hopkins Syndrome (verified by genetic testing)
GI disorder as defined below that has lasted for at least 2 years.

Sponsors & Collaborators

Gut-Brain-Axis Therapeutics Inc.
Otherlead
Pitt Hopkins Research Foundation
Othercollaborator

Study Sites (1)

Autism/Asperger's Research Group at Arizona State University

Tempe, Arizona, 85287, United States

RECRUITING

James Adams, PhdCONTACT480-818-0741 jim.adams@asu.edu

Jasmine Kirby, MSCONTACT4802343767 jkkirby1@gmail.com

James Adams, PhDPRINCIPAL INVESTIGATOR

Rosa Krajmalnik-Brown, PhDSUB INVESTIGATOR

MeSH Terms

Conditions

Pitt-Hopkins syndrome

Interventions

Vancomycinmagnesium citrateAntacids

Intervention Hierarchy (Ancestors)

GlycopeptidesGlycoconjugatesCarbohydratesPeptidesAmino Acids, Peptides, and ProteinsMolecular Mechanisms of Pharmacological ActionPharmacologic ActionsChemical Actions and UsesGastrointestinal AgentsTherapeutic Uses

Central Study Contacts

James B Adams

CONTACT

4809653316 jim.adams@asu.edu

Jasmine K Kirby

CONTACT

4802343767

Study Design

Study Type: interventional
Phase: phase 2
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

March 7, 2024

First Posted

March 20, 2024

Study Start

February 27, 2024

Actual (reported)

You may not qualify if:

Antibiotics in 2 months prior to start of treatment (topical antibiotics are allowed)
Probiotics in 2 months prior to start of treatment, or fecal transplant in last 12 months. Foods naturally containing probiotics such as yogurt are allowed.
Current severe gastrointestinal problems that require immediate hospital 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), or active malignancy or infection.
Recent or scheduled abdominal surgeries
Current participation in other clinical trials
Females who are pregnant or who are at risk of pregnancy and sexually active with a male partner without effective birth control. We will conduct a pregnancy test on all female participants 12 years and older as part of the screening and at each clinical visit.
Males who are sexually active with a female partner without highly effective birth control (IUD or birth control hormones).
Allergy or intolerance to the study medications: vancomycin, magnesium citrate, milk powder with chocolate flavoring (which are included in MTP-101P), or the antacid.
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.
Substantially decreased kidney function, as evidenced by estimated glomerular filtration rate of \<60 mL/min/1.73 m2. This is not normally reported for children on standard laboratory metabolic panels, so in those cases we will use the National Kidney Foundations Pediatric Glomerular filtration rate (GFR) Calculator to calculate the pediatric GFR based on age/height, the Blood urea nitrogen (BUN) and serum creatinine from our standard Comprehensive Metabolic Panel (CMP). \[https://www.kidney.org/professionals/kdoqi/gfr\_calculatorped\] This calculator uses the Creatinine-based "Bedside Schwartz" equation (2009) that seems to be the most commonly used calculation for this purpose.
Participants who are breastfeeding.

Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (8)

Study Arms (2)

Group A: Treatment

Group B: Placebo

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Intervention Hierarchy (Ancestors)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (8)

Study Arms (2)

Group A: Treatment

Group B: Placebo

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Intervention Hierarchy (Ancestors)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations