A Phase 2a Open-Label Study to Evaluate the Efficacy and Safety of MORF-057 in Adults With UC

Study Overview
=================
Brief Summary
-----------------
This is an open-label, single arm, multicenter, Phase 2a study evaluating the efficacy, safety, and tolerability of MORF-057 in adult patients with Moderately to Severely Active Ulcerative Colitis (UC)

Detailed Description
-----------------
The main part of this Phase 2a study will consist of 3 study periods: a Screening Period, a Treatment Period and a Safety Follow-up Period. All participants who complete the open-label Treatment Period will have the opportunity to continue their treatment in an optional 26-week Long-term Extension study after completing the Week 52 assessments.

Official Title
-----------------
A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Tolerability of MORF-057 in Adults With Moderately to Severely Active Ulcerative Colitis (EMERALD-1)

Conditions
-----------------
Inflammatory Bowel Diseases, Colitis, Ulcerative

Intervention / Treatment
-----------------
* Drug: MORF-057


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Has signs/symptoms of moderately to severely active UC for at least 3 months prior to Screening Has evidence of UC extending at least 15 cm from the anal verge Is bio-naïve or had an inadequate response, loss of response, or intolerance to other UC drugs Agrees to abide by the study guidelines and requirements Capable of giving signed informed consent Exclusion Criteria: Diagnosed with indeterminate colitis, microscopic colitis, ischemic colitis, radiation colitis, or Crohn's disease or has clinical findings suggestive of Crohn's disease Has positive findings on a subjective neurological screening questionnaire Has a concurrent, clinically significant, serious, unstable comorbidity Primary non-responder to vedolizumab or other integrin inhibitors Participation in any other interventional study or received any investigational therapy within 30 days Previous exposure to MORF-057 and/or a known hypersensitivity to drugs with a similar mechanism to MORF-057 Unable to attend study visits or comply with study procedures

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 85 Years

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

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

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

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MORF-057<br> | Drug: MORF-057<br>* MORF-057 is a small molecule that is designed to selectively inhibit integrin α4β7 and is administered orally.<br>|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline to Week 12 in the Robarts Histopathology Index (RHI) Score |  | From baseline to 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequencies and proportions for treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and TEAEs leading to study drug discontinuation overtime |  | 12 weeks | 
| Change from baseline to Week 12 in the Modified Mayo Clinic Score |  | From baseline to 12 weeks | 
| Maximum Plasma Concentration (Cmax) during single and multiple doses of MORF-057 |  | 12 weeks | 
| Time to reach Cmax (Tmax) during single and multiple doses of MORF-057 |  | 12 weeks | 
| Area under the curve (AUC) following single and multiple doses of MORF-057 |  | 12 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Ulcerative Colitis (UC), Inflammatory Bowel Disease (IBD), a4b7, Moderately to severely active ulcerative colitis, Integrin