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Phase 2 N=10 Randomized Quadruple-blind Treatment

Pilot Study of Fundamental Modification of the Gut Microbiota in the Treatment of Refractory Crohn's Disease

Crohn Disease

Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Feb 2022
Primary outcome: Primary: Change in FCP in Group 2 Participants — 1501 mcg/g — p=1.0

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Vancomycin (Drug); Neomycin (Drug); Ciprofloxacin (Drug); Polyethylene Glycol 3350 (Drug); Fluconazole (Drug)
Age
Pediatric, Adult, Older Adult · 6+ yrs
Sex
All
Sponsor
Children's Hospital of Philadelphia
Primary completion
Dec 2020

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in FCP in Group 2 Participants
1501 1.0
PRIMARY
Change in Disease Activity by Pediatric Crohn's Disease Activity Index
PRIMARY
Change in Disease Activity by Fecal Calprotectin (FCP)
SECONDARY
Change in C-reactive Protein (CRP)
SECONDARY
Safety and Tolerability of the Treatment Regimen Based on Medication Side Effects and/or Adverse Events

Summary

The purpose of this study is to determine the effect of a novel gut microbiota-targeted therapeutic regimen (bowel lavage and antibiotics with or without an antifungal) in the management of active Crohn's Disease (CD) or indeterminate colitis (IBDU) that is refractory to conventional, immunosuppressive therapy. In addition, the study will determine the effect of PEG lavage alone on fecal calprotectin and gut microbiota in patients who are undergoing a PEG lavage for clinical care.

Eligibility Criteria

Group 1

Inclusion Criteria

  • Males or females 6-18 years of age
  • Current weight >10 kg (or 22 lb)
  • Ability to swallow pills
  • Normal kidney function
  • Normal Aspartate transaminase (AST), Alanine transaminase (ALT), and alkaline phosphatase
  • Active CD or IBDU defined as PCDAI ≥ 30
  • C-Reactive Protein (CRP) ≥ 15mg/L (or 1.5mg/dL) or fecal calprotectin (FCP)>350mcg/g (within one month of enrollment)
  • Have been treated with one of the following therapies for at least 8 weeks with primary nonresponse or an initial response, followed by loss of response [LOR] (self-reported worsening of symptoms for ≥ 7 days): azathioprine, 6-mercaptopurine, methotrexate, adalimumab, certolizumab, golimumab, infliximab, natalizumab, vedolizumab, or ustekinumab **These medications must have been administered at standard, therapeutic dosages.

Exclusion Criteria

  • Known allergy or intolerance to aminoglycosides or any of the medications used in this study
  • Current use of one or more of the following medications: 5-fluorouracil, digoxin, anticoagulants, theophylline, phenytoin, probenecid, duloxetine, clozapine, sildenafil, hydrochlorothiazide, cyclosporine, hypoglycemics, terfenadine, tacrolimus, rifabutin, midazolam, and voriconazole
  • Known diagnosis of diabetes mellitus
  • Known or suspected structuring disease producing obstructive symptoms
  • Active Clostridium difficile infection
  • Prolonged QTc interval as seen on enrollment EKG
  • Current use of antibiotics
  • Starting or increasing the dose of an IBD related medication within 4 weeks of screening

Group 2

Inclusion Criteria

  • Males or females 10 years of age and older.
  • Patients undergoing a clinical GI endoscopy due to suspicion for active intestinal inflammation determined by physician global assessment (PGA).
  • Undergoing a bowel preparation as part of clinical care.
  • Parental/guardian permission (informed consent) and if appropriate, child assent.

Exclusion Criteria

  • Antibiotic use within the past 30 days.
  • Current presence of an ostomy bag.
  • Patients undergoing a non- polyethylene glycol 3350 cleanout.
  • Unwillingness to provide informed consent.
  • Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03476317). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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