Microbiota Transfer Therapy for Pitt-Hopkins Syndrome and Gastrointestinal Disorders

Overseen By James B Adams

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Gut-Brain-Axis Therapeutics Inc.

Must not be taking: Antibiotics, Probiotics

Disqualifiers: Ulcerative colitis, Crohn's disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment called Microbiota Transfer Therapy (MTT) to assist individuals with Pitt-Hopkins Syndrome who experience ongoing gut problems such as constipation, bloating, and abdominal pain. The treatment involves several steps, beginning with an antibiotic and bowel cleanse, followed by a special mix of substances to improve gut health over 12 weeks. Individuals with Pitt-Hopkins Syndrome and related gastrointestinal issues for at least two years, who have not found relief from standard treatments, might be suitable candidates for this study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you do not change your medications, supplements, diet, or therapies for at least 2 months before starting and during the trial, except for GI medications, which may be reduced if symptoms improve.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that Microbiota Transfer Therapy (MTT) has been tested in earlier studies for safety and tolerability. These studies found that patients with conditions similar to those in this trial generally tolerate MTT well. Most participants experienced few side effects, which were usually mild. Common side effects included temporary digestive issues, such as mild stomach discomfort or gas.

Although researchers are still testing this treatment, the current phase of the trial suggests there is already some evidence supporting its safety in humans. This indicates that earlier research has shown enough promise to warrant further study. Those considering joining a clinical trial for MTT should know that it has a track record of being relatively safe so far, with mostly mild side effects reported. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Pitt-Hopkins Syndrome?

Microbiota Transfer Therapy is unique because it uses the transfer of beneficial gut bacteria to address both Pitt-Hopkins Syndrome and associated gastrointestinal disorders. Unlike traditional treatments that might focus on symptom management with medications like laxatives or dietary changes, this therapy targets the underlying gut microbiome imbalance. Researchers are excited about this approach because it offers the potential for more comprehensive and lasting improvements in both neurological and gastrointestinal symptoms by directly modifying the gut environment.

What evidence suggests that Microbiota Transfer Therapy could be an effective treatment for Pitt-Hopkins Syndrome and gastrointestinal disorders?

Research shows that Microbiota Transfer Therapy (MTT), which participants in this trial may receive, might help treat stomach and digestion problems in people with Pitt-Hopkins Syndrome (PTHS). Earlier studies have found MTT promising in easing gut-related symptoms. For instance, a study on children with autism, who often have similar gut issues, showed that their symptoms improved by nearly 50% two years after receiving MTT. This therapy works by replacing harmful bacteria in the gut with healthy ones, aiding digestion and reducing problems like constipation and bloating. Although specific data for PTHS is limited, these findings suggest possible benefits for gut health in similar conditions. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

This trial is for children and adults with Pitt Hopkins Syndrome, which includes intellectual disability and gastrointestinal issues like constipation. Participants should also have a history of hyperventilation or abnormal facial expressions related to the syndrome.

Inclusion Criteria

Review of last year of medical records by the study physician

I am between 5 and 54 years old with genetically confirmed Pitt Hopkins Syndrome.

I have had a gastrointestinal disorder for at least 2 years.

Exclusion Criteria

Current participation in other clinical trials

Allergy or intolerance to the study medications: vancomycin, magnesium citrate, milk powder with chocolate flavoring, or the antacid

Tube feeding if participant requires an unusual diet such as a liquid diet with low fiber

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Placebo-Controlled Treatment

Randomized, double-blind, placebo-controlled trial with 10 days of oral vancomycin or placebo, 1 day of magnesium citrate, 4 days of high dose MTP-101P, followed by 12 weeks of lower maintenance dose of MTP-101P

14 weeks

Open-Label Observation and Cross-Over

Group A: Observation over 14 weeks. Group B: Receives the same treatment as Group A in Part 1

14 weeks

Follow-up

Participants are monitored for long-term efficacy and possible adverse effects

14 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Microbiota Transfer Therapy

Trial Overview The study tests Microbiota Transfer Therapy (MTT), involving an antibiotic called vancomycin, a bowel cleanse with magnesium citrate, followed by high then lower doses of MTP-101P with an antacid over several weeks against placebo treatments.

How Is the Trial Designed?

Group I: Group A: Treatment Experimental Treatment 4 Interventions

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

Group II: Group B: Placebo Placebo Group 4 Interventions

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

Microbiota Transfer Therapy is already approved in United States for the following indications:

Approved in United States as Microbiota Transfer Therapy for:

• Pitt-Hopkins Syndrome with gastrointestinal disorders (constipation, bloating, abdominal pain)

Who Is Running the Clinical Trial?

Gut-Brain-Axis Therapeutics Inc.

Lead Sponsor

Trials

2

Recruited

80+

Pitt Hopkins Research Foundation

Collaborator

Trials

3

Recruited

20,000+

Pitt Hopkins Research Foundation

Collaborator

Trials

3

Recruited

20,000+

Published Research Related to This Trial

A 71-year-old male patient with Parkinson's disease and severe constipation experienced significant relief after undergoing fecal microbiota transplantation (FMT), achieving normal bowel movements within 5 minutes post-procedure.

The patient's Parkinson's symptoms, particularly tremors, improved notably within a week of FMT, suggesting that gut microbiota reconstruction may offer therapeutic benefits for Parkinson's patients with gastrointestinal issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation to treat Parkinson's disease with constipation: A case report. Huang, H., Xu, H., Luo, Q., et al.[2022]

Microbiota Transplant Therapy (MTT) has shown to be relatively safe and effective in reducing gastrointestinal disorders and autism symptoms in children, as indicated by an open-label study and a two-year follow-up.

MTT resulted in significant changes to the gut microbiome structure and increased microbial diversity, suggesting a potential mechanism for its therapeutic effects in individuals with autism spectrum disorders and chronic gastrointestinal issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Microbiota transplant therapy and autism: lessons for the clinic. Adams, JB., Borody, TJ., Kang, DW., et al.[2020]

Washed microbiota transplantation (WMT) using colonic transendoscopic enteral tubing (TET) was successfully performed in 38 children with autism spectrum disorder (ASD), achieving a 100% success rate across 124 procedures.

The TET method was found to be safe, with no severe adverse events reported, and parents expressed high satisfaction with the procedure, indicating its feasibility for treating symptoms associated with ASD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Colonic Endoscopic Tubing Is Safe and Effective Approach for Washed Microbiota Transplantation in Autistic Children. Yuan, QF., Wu, HY., Chen, XY., et al.[2023]

Fecal microbiota transplantation (FMT) has shown clinical benefits in treating a wide range of dysbiosis-related diseases, with 782 studies reviewed covering 85 specific conditions, indicating its potential effectiveness beyond just gut health.

Innovative methodologies like washed microbiota transplantation (WMT) and colonic transendoscopic enteral tubing (TET) are enhancing the delivery and efficacy of FMT, suggesting a promising future for this therapy in various medical fields.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Encyclopedia of fecal microbiota transplantation: a review of effectiveness in the treatment of 85 diseases. Wang, Y., Zhang, S., Borody, TJ., et al.[2023]

Fecal microbiota transplantation (FMT) is highly effective for treating Clostridium difficile infections and shows promise for managing various extra-intestinal disorders, including metabolic diseases and neuropsychiatric conditions.

A randomized controlled trial demonstrated that FMT from lean donors improved insulin sensitivity and increased beneficial gut bacteria in individuals with metabolic syndrome, suggesting FMT's potential in treating metabolic disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation broadening its application beyond intestinal disorders. Xu, MQ., Cao, HL., Wang, WQ., et al.[2022]

Fecal microbiota transplantation (FMT) is primarily studied for conditions like Clostridium difficile infection and inflammatory bowel disease, with 84% of the 85 reports focusing on these areas, but most studies (87%) were non-randomized controlled trials.

Important methodological details about FMT, such as donor eligibility criteria and stool collection methods, are often poorly reported, which hinders the ability to replicate studies and fully understand the safety and efficacy of FMT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Methods and Reporting Studies Assessing Fecal Microbiota Transplantation: A Systematic Review. Bafeta, A., Yavchitz, A., Riveros, C., et al.[2022]

Fecal microbiota transplantation (FMT) is a safe and effective treatment for recurrent Clostridium difficile infection (CDI), and it is now recommended for this specific use.

While FMT shows promise for other gastrointestinal conditions like ulcerative colitis (UC), Crohn's disease (CD), and irritable bowel syndrome (IBS), it is not yet recommended outside of CDI due to safety and efficacy concerns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fecal microbiota transplantation for the treatment of patients with ulcerative colitis and other gastrointestinal conditions beyond Clostridium difficile infection: an update. Holleran, G., Scaldaferri, F., Ianiro, G., et al.[2018]

Fecal microbiota transplantation (FMT) is an effective method for modifying the gut microbiome and has shown promise in treating various conditions, including Clostridiodes difficile infection (CDI) and other gastrointestinal and neurological disorders.

While FMT is currently approved for CDI in Taiwan, there is a growing interest in its potential applications for other diseases, particularly in children, where data is still limited.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current and future applications of fecal microbiota transplantation for children. Chen, CC., Chiu, CH.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06321796?term=AREA%5BConditionSearch%5D(Pitt-hopkins%20syndrome%20OR%20%22%22)%20AND%20AREA%5BOverallStatus%5D(NOT_YET_RECRUITING%20OR%20RECRUITING)&rank=1

Microbiota Transfer Therapy for Children and Adults With ... The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt-Hopkins Syndrome (PTHS) and gastrointestinal ...

2. trialx.com trialx.com/clinical-trials/listings/322983/microbiota-transfer-therapy-for-children-and-adults-with-both-pitt-hopkins-syndrome-and-gastrointestinal-disorders/

Clinical Research Trial Listing ( Pitt Hopkins Syndrome ... The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt-Hopkins Syndrome (PTHS) and ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04132427?cond=pitt%20hopkins&rank=5

MTT for Children With Both Pitt Hopkins Syndrome and ... 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.

4. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT06321796/

Trial | NCT06321796 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- ...

5. pitthopkins.org pitthopkins.org/mtt-clinical-trial/

Microbiota Transfer Therapy Clinical Trial Their research noted Autism symptoms were reduced by nearly 50% two years after fecal transplant in their trial participants, and consequently ...

6. ctv.veeva.com ctv.veeva.com/study/microbiota-transfer-therapy-for-children-and-adults-with-both-pitt-hopkins-syndrome-and-gastrointest

Microbiota Transfer Therapy for Children and Adults With Both ... The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt-Hopkins Syndrome (PTHS) and ...

7. withpower.com withpower.com/trial/phase-2-gastrointestinal-diseases-1-2024-bbf5b

Microbiota Transfer Therapy for Pitt-Hopkins Syndrome ... This Phase 2 medical study run by Gut-Brain-Axis Therapeutics Inc. is evaluating whether Microbiota Transfer Therapy will have tolerable side effects ...