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Phase 3 N=138 Randomized Triple-blind Treatment

Efficacy of Alicaforsen in Pouchitis Patients Who Have Failed to Respond to at Least One Course of Antibiotics

Pouchitis

Enrolled (actual)
138
Serious AEs
4.4%
Results posted
Feb 2020
Primary outcome: Primary: Proportion of Patients With Endoscopic Remission — 3; 3 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Alicaforsen (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Atlantic Pharmaceuticals Ltd
Primary completion
Jul 2018

Outcome Measures

OutcomeResultp-value
PRIMARY
Proportion of Patients With Endoscopic Remission
3; 3
PRIMARY
Proportion of Patients With a Reduction in Relative Stool Frequency
22; 16

Summary

A Phase III, multi-centre, double-blind randomised controlled trial in subjects with chronic antibiotic refractory pouchitis. Subjects will undertake a <2 week screening period to provide baseline data and be assessed for eligibility. At the Baseline visit (Day 1) eligible subjects will be randomised on a 1:1 basis to either a) 240 mg alicaforsen enema or b) matching placebo. Study drug will be administered once nightly (on going to bed) up to and including week 6. Following the Day 1 Visit, subjects will return to the clinic for safety and efficacy assessments at Week 3, 6, 10, 18 and 26. Subjects may receive certain permitted medications as per Entry Criteria, which must remain at stable doses throughout the trial. Introduction of any new medication for pouchitis, or a dose change to an existing concomitant medication for pouchitis, other than those detailed in the protocol, will not be permitted. Clinical symptoms associated with pouchitis will be recorded daily by the patient in a diary card. Subjects will undergo endoscopic examination of their pouch (during Screening, and at Weeks 6 and 10). Where technically feasible, each endoscopy will provide at least one biopsy sample for histopathology. In addition to endoscopic, histopathologic and symptomatic assessments, Quality of Life will be assessed. Bloods for routine assessment, including haematology and biochemistry will be taken. Bloods and stool samples will be collected to evaluate relevant biomarkers.

Eligibility Criteria

Inclusion Criteria

  • Written informed consent;
  • Male or female subjects, 18 years of age who have undergone an IPAA for UC
  • History of pouchitis
  • Overall PDAI score > 7
  • Must have Chronic Antibiotic Refractory Pouchitis

Exclusion Criteria

  • Lack of effective contraception
  • Women who are pregnant or breastfeeding;
  • Strong analgesia NSAID use
  • Change in dose of the following permitted meds during screening and study: oral 5-aminosalicylate (5 ASA), Oral steroids,, Immunosuppressant therapy.
  • Rectal products
  • Biological agents: Anti-tumour necrosis factor (anti - TNF) therapy and / or vedolizumab; are not permitted within 8 weeks of the Screening Visit.
  • All other agents targeted to pouchitis, including experimental agents, must have been discontinued at least 8 weeks prior to the Screening Visit, or for a period equivalent to 5 half-lives (t½) of the agent (whichever is longer)
  • Anal sphincter dysfunction
  • Infections to cytomegalovirus or Clostridium Difficile
  • Other GI pathology (inc. intestinal malabsorption, pancreatic maldigestion etc) and differential diagnoses
  • Clinically significant and/or persistent illness; which in the investigators opinion, would exclude entry into the trial
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02525523). 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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