A Placebo-controlled Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis

Study Overview
=================
Brief Summary
-----------------
This trial is conducted in Europe. The aim of the trial is to investigate the effect of recombinant factor XIII (rFXIII) administered to subjects with mild to moderate active ulcerative colitis (UC).

Official Title
-----------------
A Multicenter, Randomised, Double-blind, Placebo-controlled, Multiple-dose Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis

Conditions
-----------------
Inflammation, Ulcerative Colitis

Intervention / Treatment
-----------------
* Drug: catridecacog
* Drug: placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Diagnosis of ulcerative colitis for at least 3 months from the time of initial diagnosis. The diagnosis must have been confirmed by historical endoscopy and histology. The severity of disease must have been confirmed by endoscopy at screening Currently receiving oral aminosalicylates at approved doses of at least 2g/day for at least 6 weeks. Doses of oral aminosalicylates should be stable for at least two weeks prior to dosing (Visit 2) Exclusion Criteria: Diagnosis of UC limited to the rectum (ulcerative proctitis only, defined as less than 15 cm from the anal verge) Requiring hospitalisation for current episode of severe UC Use of biologic therapies for the treatment of UC within 12 weeks prior to dosing (Visit 2) Treatment failures to anti-tumour necrosis factor-alfa (anti-TNF-a) agents (e.g. infliximab, adalimumab) Use of immunosuppressant agents (e.g. azathioprine) within 4 weeks prior to dosing (Visit 2) Use of corticosteroids (oral, intravenous (i.v.), intramuscular (i.m.), or rectal ) within 14 days prior to dosing (Visit 2) Use of enemas (corticosteroid or aminosalicylate) within 14 days prior to screening (Visit 1) Use of cyclosporine, tacrolimus, D-penicillamine, leflunomide, methotrexate, mycophenolate mofetil, or thalidomide within 4 weeks prior to dosing (Visit 2) Currently receiving total parenteral nutrition

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 64 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFXIII<br> | Drug: catridecacog<br>* Catridecacog (recombinant factor XIII, rFXIII) will be administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week at a dose of 35 IU/kg<br>* Other names: recombinant factor XIII;|
| Active Comparator: Placebo<br> | Drug: placebo<br>* Placebo will be administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Endoscopic Remission Defined as a Modified Baron Score of 0 | The primary endpoint was the binary variable (responder vs. non-responder) where responders were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as non-responders. | At week 8 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Remission (Clinical and Endoscopic) | Analysis of responders defined by a clinical component of: ulcerative colitis disease activity index (UC-DAI) score of less than or equal to 1 with 0 for rectal bleeding and 0 for stool frequency and an endoscopic component of: no mucosal friability (modified Baron score less than or equal to 1). | At Week 8 | 
| Number of Adverse Events (AEs) | Number of adverse events reported from the first trial-related activity, after the subject was exposed to the trial drug, until the end of the post-treatment follow-up period. | Week 0 to 10 | 
| Clearance (CL) of rFXIII | The volume of plasma cleared of the drug per unit time. | Samples were collected before and up to 72 hours after the first dose of rFXIII. | 
| Maximum Concentration (Cmax) of rFXIII | The peak plasma concentration of the drug after dose administration. | Samples were collected before and up to 72 hours after the first dose of rFXIII. |