FMT as a Treatment for Severe Motility Disorder

NCT04373252 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: 18-2327
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Many patients that are treated with anorectal malformations are fecally incontinent for life. A Bowel Management Program has been developed to help these patients by creating a daily enema regimen to keep them artificially clean of stool in the underwear. Due to the high success rate of the program, many patients who suffer from fecal incontinence due to other reasons such as, spina bifida, sacrococcygeal teratoma and sacral agenesis are referred to the program. A new issue is emerging with a group of patients that no longer obtain effective results from their daily enemas, even though they have worked successfully for years. These same patients are presenting with a narrow, spastic left colon and remarkably dilated right colon. Our hypothesis is that prolonged enema administration negatively impacts the microbiota of the colon causing the lack of response from enema administration. The purpose of this study is to restore the normal flora of the colon by fecal microbiota transplant (FMT) which we believe will improve responsiveness to enemas. By restoring colonic flora, patients will again become responsive to daily enemas and regain successful bowel management.

Conditions

Severe Motility Disorder

Outcome Measures

Primary Outcomes (1)

• Patients become responsive to daily enema treatments again

  The primary outcome measure for this study will be the results reported in the daily journals which will identify if patients become responsive to enema treatments again (administration and complete evacuation within a one-hour period) and are able to remain clean in the underwear for 24 hours.

  1 year

Secondary Outcomes (1)

• Microbiota Analysis

  10 days

Study Arms (1)

Fecal Microbiota Transplant

EXPERIMENTAL

All participants will receive two fecal microbiota transplants one week apart. The transplant will occur via antegrade enema and the enema transplant material will be provided by OpenBiome, the product used is FMT Lower Delivery (FMP 30).

Biological: FMT Lower Delivery (FMP 30)

Interventions

FMT Lower Delivery (FMP 30) BIOLOGICAL

Participants will receive two fecal microbiota transplants via antegrade enema, one week apart.

Fecal Microbiota Transplant

Eligibility Criteria

• Age: 3 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• The study population to be enrolled in this study will include patients 18 years of age and older, with a Malone (appendicostomy) or with a cecostomy, that have received daily enemas for the treatment of fecal incontinence with prior good results for months to years. However, now these patients report not having an acceptable enema response (up to 4 hours for evacuation) despite numerous attempts to adjust the enema additives. The patients typically display a narrow, spastic left colon and a dilated right colon on contrast enema. A minimum of 10 adult subjects will be enrolled first and followed for safety and efficacy outcomes for at least one month. After the initial 10 adult subjects, there will be a pause in enrollment to allow for a formal review of the adult safety and efficacy data by the primary investigator. At this time a request will be made to open enrollment of this study up to the pediatric population as well as adults.

You may not qualify if:

• Any patient who does not meet the above criteria will be excluded from this study. Also excluded: pregnant women, women of child-bearing potential who are unwilling to use an effective form of contraception for 30 days after FMT, women who are breastfeeding, subjects with end stage liver disease or cirrhosis, subjects with a life expectancy of less than 6 months, subjects with an absolute neutrophil count \<500 cells/µL and those with severe food allergies.

Sponsors & Collaborators

• University of Colorado, Denver: lead
• OpenBiome: collaborator

Study Officials

• Andrea Bischoff, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2020
• First Posted: May 4, 2020
• Study Start: July 1, 2021
• Primary Completion: July 1, 2021
• Study Completion: July 1, 2021
• Last Updated: May 6, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share