Trial Outcomes & Findings for Pilot Study of Fundamental Modification of the Gut Microbiota in the Treatment of Refractory Crohn's Disease (NCT NCT03476317)

Results Overview

Change in fecal calprotectin between stool samples collected at baseline and day 12 after procedure in Group 2 participants.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

change from baseline to day 12

Results posted on

2022-02-09

Participant Flow

Participant milestones

Participant milestones
Measure	Group 1-Fluconazole Vancomycin oral suspension four times daily (Day 1-14), plus neomycin orally three times daily (Days 1-3), plus ciprofloxacin orally twice daily (Day 4-14), plus Polyethylene Glycol 3350 (Miralax) dissolved in Gatorade on day 2, plus fluconazole orally once daily (Day 1-14). Vancomycin: Oral suspension 4 times daily (Day 1-14) Neomycin: Oral three times daily (Days 1-3) Ciprofloxacin: Oral twice daily (Days 4-14) Polyethylene Glycol 3350: Dissolved in Gatorade on day 2 Fluconazole: Orally once daily (Day 1-14)	Group 1-Placebo Vancomycin oral suspension four times daily (Day 1-14), plus neomycin orally three times daily (Days 1-3), plus ciprofloxacin orally twice daily (Day 4-14), plus Polyethylene Glycol 3350 (Miralax) dissolved in Gatorade on day 2, plus placebo. Vancomycin: Oral suspension 4 times daily (Day 1-14) Neomycin: Oral three times daily (Days 1-3) Ciprofloxacin: Oral twice daily (Days 4-14) Polyethylene Glycol 3350: Dissolved in Gatorade on day 2	Group 2 Collect stool samples for calprotectin in patients undergoing colonoscopy for clinical care to evaluate effect of bowel lavage alone on calprotectin.
Overall Study STARTED	0	0	10
Overall Study COMPLETED	0	0	10
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group 1-Fluconazole Vancomycin oral suspension four times daily (Day 1-14), plus neomycin orally three times daily (Days 1-3), plus ciprofloxacin orally twice daily (Day 4-14), plus Polyethylene Glycol 3350 (Miralax) dissolved in Gatorade on day 2, plus fluconazole orally once daily (Day 1-14). Vancomycin: Oral suspension 4 times daily (Day 1-14) Neomycin: Oral three times daily (Days 1-3) Ciprofloxacin: Oral twice daily (Days 4-14) Polyethylene Glycol 3350: Dissolved in Gatorade on day 2 Fluconazole: Orally once daily (Day 1-14)	Group 1-Placebo Vancomycin oral suspension four times daily (Day 1-14), plus neomycin orally three times daily (Days 1-3), plus ciprofloxacin orally twice daily (Day 4-14), plus Polyethylene Glycol 3350 (Miralax) dissolved in Gatorade on day 2, plus placebo. Vancomycin: Oral suspension 4 times daily (Day 1-14) Neomycin: Oral three times daily (Days 1-3) Ciprofloxacin: Oral twice daily (Days 4-14) Polyethylene Glycol 3350: Dissolved in Gatorade on day 2	Group 2 n=10 Participants Collect stool samples for calprotectin in patients undergoing colonoscopy for clinical care to evaluate effect of bowel lavage alone on calprotectin.	Total n=10 Participants Total of all reporting groups
Age, Categorical <=18 years	—	—	9 Participants n=10 Participants	9 Participants n=10 Participants
Age, Categorical Between 18 and 65 years	—	—	1 Participants n=10 Participants	1 Participants n=10 Participants
Age, Categorical >=65 years	—	—	0 Participants n=10 Participants	0 Participants n=10 Participants
Age, Continuous	—	—	16.5 years n=10 Participants	16.5 years n=10 Participants
Sex: Female, Male Female	—	—	2 Participants n=10 Participants	2 Participants n=10 Participants
Sex: Female, Male Male	—	—	8 Participants n=10 Participants	8 Participants n=10 Participants
Race and Ethnicity Not Collected	—	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	—	—	10 participants n=10 Participants	10 participants n=10 Participants
Calprotectin	—	—	1254.5 mcg/g n=10 Participants	1254.5 mcg/g n=10 Participants

PRIMARY outcome

Timeframe: change from baseline to day 12

Population: Group 1 study was not done.

Change in fecal calprotectin between stool samples collected at baseline and day 12 after procedure in Group 2 participants.

Outcome measures

Outcome measures
Measure	Group 1-Fluconazole Vancomycin oral suspension four times daily (Day 1-14), plus neomycin orally three times daily (Days 1-3), plus ciprofloxacin orally twice daily (Day 4-14), plus Polyethylene Glycol 3350 (Miralax) dissolved in Gatorade on day 2, plus fluconazole orally once daily (Day 1-14). Vancomycin: Oral suspension 4 times daily (Day 1-14) Neomycin: Oral three times daily (Days 1-3) Ciprofloxacin: Oral twice daily (Days 4-14) Polyethylene Glycol 3350: Dissolved in Gatorade on day 2 Fluconazole: Orally once daily (Day 1-14)	Group 1-Placebo Vancomycin oral suspension four times daily (Day 1-14), plus neomycin orally three times daily (Days 1-3), plus ciprofloxacin orally twice daily (Day 4-14), plus Polyethylene Glycol 3350 (Miralax) dissolved in Gatorade on day 2, plus placebo. Vancomycin: Oral suspension 4 times daily (Day 1-14) Neomycin: Oral three times daily (Days 1-3) Ciprofloxacin: Oral twice daily (Days 4-14) Polyethylene Glycol 3350: Dissolved in Gatorade on day 2	Group 2 n=10 Participants Collect stool samples for calprotectin in patients undergoing colonoscopy for clinical care to evaluate effect of bowel lavage alone on calprotectin.
Change in FCP in Group 2 Participants	—	—	1501 mcg/g Interval 271.0 to 3000.0

PRIMARY outcome

Timeframe: Baseline, Day 15

Population: This outcome was specific to Group 1 and we did not enroll patients into Group1as an identical study was simultaneously enrolling adult patients and results from the adult study were sufficient to make a conclusion without enrolling children.

The primary endpoint will be the change in disease activity, as measured by Pediatric Crohn's Disease Activity Index (PCDAI) score, between the enrollment visit and Day 15. All participants who withdraw for any reason prior to day 15 will be considered treatment failures. The Pediatric Crohn's Disease Activity Index is a clinical score which takes into account general well being, degree of abdominal pain, number of liquid stools per day, abdominal physical examination, and Crohn's disease complications.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Baseline, Day 15

Population: This outcome was specific to Group 1 and Group 1 study was not completed.

The second primary outcome measure will be the change in disease activity, as measured by fecal calprotectin, between the enrollment visit and day 15. The fecal calprotectin is a stool test which measures intestinal inflammation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Day 15

Population: This outcome was specific to Group 1 and Group 1 study was not completed.

A secondary outcome measure will be the change in C-reactive protein between the enrollment visit and day 15. The C-reactive protein is a blood test which measures systemic inflammation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 105 days

Population: This outcome is specific to Group 1 and Group 1 study was not completed.

Outcome measures

Outcome data not reported

Adverse Events

Group 1-Fluconazole

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Group 1-Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Group 2

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Lindsey Albenberg, DO

Children's Hospital of Philadelphia

Phone: 267-426-0139

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place