Safety and Efficacy of QBECO in Moderate to Severe Ulcerative Colitis

Study Overview
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Brief Summary
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The purpose of this open-label, dose-ranging, exploratory study is to evaluate the safety, tolerability, compliance, mechanism of action and efficacy of QBECO site specific immunomodulation for the induction of clinical response and remission in subjects with moderate to severe ulcerative colitis

Official Title
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An Open Label, Dose Ranging, Exploratory Study to Evaluate the Safety, Tolerability, Compliance, Mechanism of Action and Efficacy of QBECO Site Specific Immunomodulation for the Induction of Clinical Response and Remission in Subjects With Moderate to Severe Ulcerative Colitis

Conditions
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Ulcerative Colitis

Intervention / Treatment
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* Biological: QBECO SSI


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female subjects who have reached age of majority Willing to learn and able to self-administer study drug Diagnosis of UC established at least 6 months before screening visit, by clinical and endoscopic evidence. Currently experiencing moderate to severe active UC defined as a Mayo score of 6-12 (inclusive) at Screening. Endoscopic evidence of active mucosal disease, as assessed by flexible sigmoidoscopy, with an Endoscopic Finding Sub-score of ≥2 at Screening. Rectal Bleeding Sub-score of ≥1 at Screening. Physician's Global Assessment Sub-score of ≥2 at Screening. Male/female subjects who agree to practice effective methods of contraception Exclusion Criteria: History of colonic or rectal surgery other than hemorrhoid surgery or appendectomy Currently receiving total parenteral nutrition Disease limited to ulcerative proctitis Diagnosed with Crohn's disease, indeterminate colitis, microscopic colitis or, ischemic or infectious colitis Known or suspected hypersensitivity to any component of the product Known human immunodeficiency virus (HIV) infection or other immunosuppressive disorder Concurrently participating in another study or receiving other experimental or investigational therapies within past 3 months Females who are currently pregnant or lactating Any history of malignancy. Exceptions may apply for cervical cancer and some forms of skin cancer

Ages Eligible for Study
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Minimum Age: 19 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: QBECO SSI 0.02 mL<br>0.02 mL administered subcutaneously, every other day for 16 weeks. After completion of the initial 16-week fixed dose treatment period, subjects deemed to be responders will be randomized to one of three maintenance dosing schedules (every second day, weekly, no treatment) for an additional 36 weeks. | Biological: QBECO SSI<br>* QBECO Site Specific Immunomodulators<br>|
| Experimental: QBECO SSI 0.05 mL<br>0.05 mL administered subcutaneously, every other day for 16 weeks. After completion of the initial 16-week fixed dose treatment period, subjects deemed to be responders will be randomized to one of three maintenance dosing schedules (every second day, weekly, no treatment) for an additional 36 weeks. | Biological: QBECO SSI<br>* QBECO Site Specific Immunomodulators<br>|
| Experimental: QBECO SSI 0.1 mL<br>0.1 mL administered subcutaneously, every other day for 16 weeks. After completion of the initial 16-week fixed dose treatment period, subjects deemed to be responders will be randomized to one of three maintenance dosing schedules (every second day, weekly, no treatment) for an additional 36 weeks. | Biological: QBECO SSI<br>* QBECO Site Specific Immunomodulators<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment emergent AEs and clinically significant changes or abnormalities from physical examinations, vital signs and laboratory results (composite) |  | Day 1 to Week 56 | 
| Proportion of subjects in clinical remission | Mayo score ≤ 2 points, with no individual subscore >1 | Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of subjects in clinical remission | Mayo score ≤ 2 points, with no individual subscore >1 | Week 16 | 
| Proportion of subjects with a clinical response | A decrease of the Mayo score from screening of at least 3 points and at least 30%, AND either a decrease from the pre-treatment screening sub-score for rectal bleeding of at least 1 point, OR rectal bleeding sub-score of 0 or 1 | Week 16 | 
| Proportion of subjects with a clinical response | A decrease of the Mayo score from screening of at least 3 points and at least 30%, AND either a decrease from the pre-treatment screening sub-score for rectal bleeding of at least 1 point, OR rectal bleeding sub-score of 0 or 1 | Week 52 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Inflammatory Bowel Disease, Ulcerative Colitis