Pitt Hopkins SyndromePhase 2NCT06321796
Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
A Phase 2 study testing MTP-101P, Vancomycin and Magnesium Citrate in people with Pitt Hopkins Syndrome, run by Gut-Brain-Axis Therapeutics Inc. 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 (constipation, bloating, abdominal pain). Read more about this study
- Who is it for?
- Ages 5 to 55 years
- Conditions the study is for
- Pitt Hopkins Syndrome
- What is being tested?
- MTP-101P, Vancomycin and Magnesium Citrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Feb 2024to May 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 5 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- No changes in medications, supplements, diet, or therapies in the 2 months prior to start of treatment, and no intention to change treatments during Part 1 (all participants) and Part 2 (group B) of the clinical trial. The only exception is GI medications, which may be reduced if symptoms reduce, and any changes during the study will be documented.
- Review of last year of medical records by the study physician.
- At least two previous trials of "standard of care" GI treatments that did not alleviate GI symptoms (constipation, diarrhea, bloating, gas, reflux, and/or abdominal pain). Standard of care treatments include laxatives, stool softeners, enemas, suppositories, or similar medications.
Who cannot join
- 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.
- Tube feeding may be an exclusion criterion if the participant requires an unusual diet such as a liquid diet with low fiber.
- 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.
Show all 15Show fewer
- 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.
- Clinically significant abnormalities at baseline on the blood safety tests, and confirmed on a second test. The tests include Comprehensive Metabolic Panel, and Complete Blood Count with Differential. Note that some abnormalities may occur due to PTHS, so only those likely to significantly increase risk in this study would be grounds for exclusion, at the discretion of the study physician. See detailed discussion at the end of this section on Interpreting Laboratory Results. re. Eligibility for Admission to Study.
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Autism/Asperger's Research Group at Arizona State University
Tempe, Arizona, 85287
James Adams, Phd, 480-818-0741, jim.adams@asu.edu
Jasmine Kirby, MS, 4802343767, jkkirby1@gmail.com
About this study
Official title: Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders: MTP-101 P
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt-Hopkins Syndrome (PTHS) and gastrointestinal problems (constipation, bloating, abdominal pain). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using magnesium citrate, followed by 4 days of high dose MTP-101P with an antacid, followed by 12 weeks of a lower maintenance dose of MTP-101P with an antacid.
Read the detailed description
For children 5-17 years old and adults 18-55 years old with Pitt-Hopkins Syndrome (PTHS) and Gastrointestinal (GI) problems who have attempted two standard-of-care (SOC) GI treatments with no alleviation of symptoms. This is a Phase 2 clinical trial that will evaluate the safety, tolerability, and efficacy of a powder version of Microbiota Transfer Therapy (MTT) called MTP-101P. 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 4 days of initial high dose of MTP-101P taken daily 5 minutes after antacid, and then 12 weeks of a lower maintenance dose of MTP-101P taken daily 5 minutes after an antacid.
Group A: Real Treatment vs. Group B: Placebo vancomycin, real magnesium citrate, placebo MTP-101P, real antacid.
Part 2: Open-Label Observation and Cross-Over (14 weeks) Group A: Observation over the next 14 weeks (no additional treatment). Group A completes the study at the end of part 2.
Group B: They will receive the same treatment that Group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, and an initial high dose of MTP-101P for 4 days taken daily 5 minutes after an antacid, and then a lower dose of MTP-101P for 12 weeks taken daily 5 minutes after antacid.
Part 3: Follow Up Group B: There will be a follow-up evaluation 14 weeks post-treatment after the end of Part 2, to assess long-term efficacy and possible adverse effects.
- Conditions
- Pitt Hopkins Syndrome
- Being tested
- MTP-101P, Vancomycin, Magnesium Citrate, Antacid, Placebo MTP-101P
- 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.
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
VancomycinCombination product
Oral vancomycin is an antibiotic used for treating C. difficile-associated diarrhea and staphylococcal-induced enterocolitis.
Duration: 10 Days
Magnesium CitrateCombination product
Magnesium citrate is a common over-the-counter laxative and bowel cleanse.
Duration: 1 Day
AntacidCombination product
The antacid will be aluminum/magnesium hydroxide, 1x/day, 5 minutes before the MTP-101P. Each 5 ml consists of Aluminum Hydroxide - 200 mg, Magnesium Hydroxide - 200 mg, and Simethicone - 20 mg. If some participants cannot tolerate the aluminum/magnesium hydroxide, we will allow calcium carbonate alternatives equivalent in acid neutralizing capacity.
Duration: 12.5 weeks
Placebo MTP-101PCombination product
The same packaging will be used as for MTP-101P, but the sachets will contain a freeze-dried mixture of normal saline containing 10% Trehalose.
Duration: 12.5 weeks
Placebo VancomycinCombination product
Same packaging and liquid carrier and flavoring used as for the oral vancomycin, but no vancomycin.
Duration: 10 days
| Study group | Role | What they receive |
|---|---|---|
| Group A: Treatment | Experimental | Part 1: Blinded Treatment (14 weeks)
Vancomycin, Magnesium Citrate, Antacid, MTP-101P Receives: Combination Product: MTP-101P; Combination Product: Vancomycin; Combination Product: Magnesium Citrate; Combination Product: Antacid |
| Group B: Placebo | Placebo comparator | Part 1: Blinded Placebo (14 weeks)
Placebo Vancomycin, Real Magnesium Citrate, Real Antacid, Placebo MTP-101P Receives: Combination Product: Magnesium Citrate; Combination Product: Antacid; Combination Product: Placebo MTP-101P; Combination Product: Placebo Vancomycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Days 0 to 99 of treatment
Secondary outcomes (8)
Gastrointestinal Symptom Rating Scale (GSRS)
An assessment of GI symptoms based on 15 questions, which are then scored in five domains (abdominal pain, reflux, indigestion, diarrhea, and constipation). Symptoms are rated on a 7-point Likert scale including no discomfort at all (1), slight discomfort (2), mild discomfort (3), moderate discomfort (4), moderately severe discomfort (5), severe discomfort (6), and very severe discomfort (7).
Measured: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Clinical Global Impression - Gastrointestinal (CGI-GI)
This assessment is conducted by the study physician and assess the severity of GI symptoms on a 7-point scale. The severity scale rates the severity of the patient's illness including normal, not at all ill (1), borderline mentally ill (2), mildly ill (3), moderately ill (4), markedly ill (5), severely ill (6), among the most extremely ill patients (7).
Measured: Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment)
Clinical Global Impression - Pitt Hopkins (CGI-PTHS)
This assessment is conducted by the study physician and assess the severity of Pitt Hopkins Symptoms on a 7-point scale. The severity scale rates the severity of the patient's illness including normal, not at all ill (1), borderline mentally ill (2), mildly ill (3), moderately ill (4), markedly ill (5), severely ill (6), among the most extremely ill patients (7).
Measured: Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment).
Parent Global Impressions - Pitt Hopkins: Change in Symptoms (PGI-PTHS-2-Change)
An assessment of Pitt Hopkins Syndrome symptoms that rates the change in symptom severity since the start of treatment. Symptom severity is rated on a 7-point scale including much worse (-3), somewhat worse (-2), slightly worse (-1), no change (0), slightly better (1), somewhat better (2), and much better (3).
Measured: End of Treatment (After 95 days of treatment)
Gastrointestinal Symptoms common in Pitt Hopkins (GI-PTHS)
Assess common symptoms in Pitt Hopkins, including 2 questions about toilet training, a question about GI feeding tubes, and 7 questions about current GI symptom severity including constipation, diarrhea, gas, bloating, pain, reflux/heartburn/regurgitation and vomiting. Symptoms are rated on a 7-point scale from none to extremely severe.
Measured: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Revised Face Legs Activity Crying Consolability Pain Questionnaire (FLACC)
Modified from the original FLACC for children with cognitive impairment (Malviya et al., 2006). Assess pain in five areas (Face, Legs, Activity, Crying, Consolability) using a three-point scale for each area, resulting in a total FLACC pain scale ranging from zero to 10.
Measured: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Benefit
Assess the effectiveness of MTT on overall benefit (no benefit (0)/slight benefit(1)/moderate benefit (2)/good benefit (3)/great benefit (4)).
Measured: End of Treatment (After 95 days of treatment)
National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Adverse E
Assess the effectiveness of MTT on overall adverse effects of treatment (no adverse effects (0)/mild adverse effects (1)/moderate adverse effects (2)/severe adverse effects (3)).
Measured: End of Treatment (After 95 days of treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2024
- Study start27 Feb 2024
- Primary completionFeb 2025 (estimated)
- Study completionMay 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pitt Hopkins SyndromeStudies of the same condition, open and under way.
- 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-Blinded, Placebo-Controlled Treatment (14 weeks) Part 2: Open-Label Observation and Cross-over or Treatment Part 3: Follow Up (Group B Only)
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gut-Brain-Axis Therapeutics Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Pitt Hopkins Research Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06321796
- Sponsor's protocol ID
- WCG IRB Protocol #20235847
- Status last verified
- Mar 2024
- First submitted
- 7 Mar 2024
- Last updated
- 20 Mar 2024
- Results posted
- No
- 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 20 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders: MTP-101 P
- ClinicalTrials.gov ID
- NCT06321796
- Acronym / sponsor ID
- WCG IRB Protocol #20235847
- Conditions
- Pitt Hopkins Syndrome
- Keywords
- Microbiota Transfer Therapy, Fecal Transplant, Fecal Microbiota Transplant, Intestinal Microbiota
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Gut-Brain-Axis Therapeutics Inc.University, hospital or other
- Collaborators
- Pitt Hopkins Research Foundation
- Study start
- 27 Feb 2024 (actual)
- Primary completion
- Feb 2025 (estimated)
- Study completion
- May 2025 (estimated)
- First posted
- 20 Mar 2024 (actual)
- Results
- Not posted in the source record
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 (constipation, bloating, abdominal pain). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using magnesium citrate, followed by 4 days of high dose MTP-101P with an antacid, followed by 12 weeks of a lower maintenance dose of MTP-101P with an antacid.
Detailed description
For children 5-17 years old and adults 18-55 years old with Pitt-Hopkins Syndrome (PTHS) and Gastrointestinal (GI) problems who have attempted two standard-of-care (SOC) GI treatments with no alleviation of symptoms. This is a Phase 2 clinical trial that will evaluate the safety, tolerability, and efficacy of a powder version of Microbiota Transfer Therapy (MTT) called MTP-101P. 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 4 days of initial high dose of MTP-101P taken daily 5 minutes after antacid, and then 12 weeks of a lower maintenance dose of MTP-101P taken daily 5 minutes after an antacid.
Group A: Real Treatment vs. Group B: Placebo vancomycin, real magnesium citrate, placebo MTP-101P, real antacid.
Part 2: Open-Label Observation and Cross-Over (14 weeks) Group A: Observation over the next 14 weeks (no additional treatment). Group A completes the study at the end of part 2.
Group B: They will receive the same treatment that Group A received in part 1. This includes 10 days of vancomycin, magnesium citrate, and an initial high dose of MTP-101P for 4 days taken daily 5 minutes after an antacid, and then a lower dose of MTP-101P for 12 weeks taken daily 5 minutes after antacid.
Part 3: Follow Up Group B: There will be a follow-up evaluation 14 weeks post-treatment after the end of Part 2, to assess long-term efficacy and possible adverse effects.
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-Blinded, Placebo-Controlled Treatment (14 weeks) Part 2: Open-Label Observation and Cross-over or Treatment Part 3: Follow Up (Group B Only)
| Arm | Type | Receives |
|---|---|---|
| Group A: Treatment | Experimental | Combination Product: MTP-101P, Combination Product: Vancomycin, Combination Product: Magnesium Citrate, Combination Product: AntacidPart 1: Blinded Treatment (14 weeks) Vancomycin, Magnesium Citrate, Antacid, MTP-101P |
| Group B: Placebo | Placebo comparator | Combination Product: Magnesium Citrate, Combination Product: Antacid, Combination Product: Placebo MTP-101P, Combination Product: Placebo VancomycinPart 1: Blinded Placebo (14 weeks) Placebo Vancomycin, Real Magnesium Citrate, Real Antacid, Placebo MTP-101P |
Who may qualify
- Ages
- 5 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
- No changes in medications, supplements, diet, or therapies in the 2 months prior to start of treatment, and no intention to change treatments during Part 1 (all participants) and Part 2 (group B) of the clinical trial. The only exception is GI medications, which may be reduced if symptoms reduce, and any changes during the study will be documented.
- Review of last year of medical records by the study physician.
- At least two previous trials of "standard of care" GI treatments that did not alleviate GI symptoms (constipation, diarrhea, bloating, gas, reflux, and/or abdominal pain). Standard of care treatments include laxatives, stool softeners, enemas, suppositories, or similar medications.
Key exclusion criteria (15 listed)
- 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.
- Tube feeding may be an exclusion criterion if the participant requires an unusual diet such as a liquid diet with low fiber.
- Current severe gastrointestinal problems that require immediate hospital 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 |
|---|---|---|
| MTP-101PMTP-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 | Combination product | Group A: Treatment |
| VancomycinOral vancomycin is an antibiotic used for treating C. difficile-associated diarrhea and staphylococcal-induced enterocolitis. Duration: 10 Days | Combination product | Group A: Treatment |
| Magnesium CitrateMagnesium citrate is a common over-the-counter laxative and bowel cleanse. Duration: 1 Day | Combination product | Group A: Treatment, Group B: Placebo |
| AntacidThe antacid will be aluminum/magnesium hydroxide, 1x/day, 5 minutes before the MTP-101P. Each 5 ml consists of Aluminum Hydroxide - 200 mg, Magnesium Hydroxide - 200 mg, and Simethicone - 20 mg. If some participants cannot tolerate the aluminum/magnesium hydroxide, we will allow calcium carbonate alternatives equivalent in acid neutralizing capacity. Duration: 12.5 weeks | Combination product | Group A: Treatment, Group B: Placebo |
| Placebo MTP-101PThe same packaging will be used as for MTP-101P, but the sachets will contain a freeze-dried mixture of normal saline containing 10% Trehalose. Duration: 12.5 weeks | Combination product | Group B: Placebo |
| Placebo VancomycinSame packaging and liquid carrier and flavoring used as for the oral vancomycin, but no vancomycin. Duration: 10 days | Combination product | Group B: Placebo |
Primary outcome
Daily Stool Record (DSR)
Time frame: Baseline (for 14 days pre-treatment) vs End of Treatment (Days 82-95 of treatment).
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.
Safety Measures (Adverse Events)
Time frame: Days 0 to 99 of treatment
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.
8 secondary outcomes
Gastrointestinal Symptom Rating Scale (GSRS)
Time frame: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Clinical Global Impression - Gastrointestinal (CGI-GI)
Time frame: Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment)
Clinical Global Impression - Pitt Hopkins (CGI-PTHS)
Time frame: Baseline (pre-treatment) vs End of Treatment (After 99 days of treatment).
Parent Global Impressions - Pitt Hopkins: Change in Symptoms (PGI-PTHS-2-Change)
Time frame: End of Treatment (After 95 days of treatment)
Gastrointestinal Symptoms common in Pitt Hopkins (GI-PTHS)
Time frame: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
Revised Face Legs Activity Crying Consolability Pain Questionnaire (FLACC)
Time frame: Baseline (pre-treatment) vs End of Treatment (After 95 days of treatment)
National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Benefit
Time frame: End of Treatment (After 95 days of treatment)
National Survey on treatment Effectiveness for Autism (NSTEA) - Overall Adverse E
Time frame: End of Treatment (After 95 days of treatment)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2024.
- Individual participant dataSharing planned: No
Related trials for Pitt Hopkins Syndrome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 1
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
Pitt Hopkins Syndrome
RecruitingPhase 1/2
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Pitt Hopkins Syndrome
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
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.
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 MTP-101P, Vancomycin and Magnesium Citrate in people with Pitt Hopkins Syndrome, run by Gut-Brain-Axis Therapeutics Inc. 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 15 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: status not confirmed, as of the sponsor's last update on 20 Mar 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact James B Adams (4809653316, jim.adams@asu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Daily Stool Record (DSR) (time frame: Baseline (for 14 days pre-treatment) vs End of Treatment (Days 82-95 of treatment).). 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 20 Mar 2024.