SC12267 (4SC-101) for Treatment of Patients With Inflammatory Bowel Disease

Study Overview
=================
Brief Summary
-----------------
This is an exploratory, open-label, uncontrolled, multi-center, 1-arm study conducted at 16 sites in Bulgaria, Germany and Romania. A total of ca. 30 - 35 patients will receive SC12267 (4SC-101) tablets, 35 mg once daily for 12 weeks. First of all the patients will undergo a screening period of 1 week and a follow-up visit will be performed 4 weeks after study drug discontinuation or earlier in case of relapse during follow up. Total study duration will be up to 36 (+2) weeks. There will be 8 study visits: one screening visit, 6 visits during the treatment period and one follow-up visit. In addition, four telephone visits will be performed at Week 6, Week 10, Week 20 (+1) and Week 36 (+2). The duration of the entire study (first patient in till last patient out) is expected to be about 13 months.

Official Title
-----------------
Exploratory, Open-label Study to Demonstrate Efficacy, Safety and Tolerability of SC12267 (35 mg) in Patients With Inflammatory Bowel Disease (Crohn's Disease and Ulcerative Colitis)

Conditions
-----------------
Inflammatory Bowel Disease (IBD)

Intervention / Treatment
-----------------
* Drug: SC12267 (4SC-101)


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Criteria regarding Crohn´s Disease: Established diagnosis of CD, confirmed by standard criteria (e.g. endoscopy, ultrasound, X-ray) Patients must be in clinical remission (Crohn's Disease Activity Index [CDAI] <150 points) on steroid therapy for at least 2 weeks Confirmed steroid-dependency of CD: patients who are either unable to taper steroids completely within 3 months of starting steroids, without recurrent active disease, or who have a relapse within 2 months of stopping steroids Individual threshold* dose of previous relapses should be equal or less than 20 mg/day Prednisolone or equivalent steroid dose Patients with stable glucocorticosteroid therapy between 20 and 40 mg/day Prednisolone or equivalent steroid dose for the previous week Criteria regarding Ulcerative Colitis: Established diagnosis of UC, confirmed by standard criteria (e.g. endoscopy, ultrasound, X-ray) Patients must be in clinical remission (Clinical Activity Index [CAI] <4 points) on steroid therapy for at least 2 weeks Confirmed steroid-dependency of UC: patients who are either unable to taper steroids completely within 3 months of starting steroids, without recurrent active disease, or who have a relapse within 2 months of stopping steroids Individual threshold* dose of previous relapses should be equal or less than 20 mg/day Prednisolone or equivalent steroid dose Patients with stable glucocorticosteroid therapy between 20 and 40 mg/day Prednisolone or equivalent steroid dose for the previous week (* The threshold dose is that dose at which the patient experienced the relapses) Criteria regarding general requirements: Men and women, 18 to 70 years of age Written informed consent Negative pregnancy test at screening in females of child-bearing potential Males willing to use condoms or to be sexually abstinent Use of appropriate contraceptive methods for females of childbearing potential one month before, throughout the course of the study and one month after study termination. This must be a combination of the following: a highly effective method of first choice = a method with a low failure rate (i.e. less than 1% per year) like sexual abstinence, combined oral contraceptives, implants, injectables, some Intra Uterine Devices (IUDs), vasectomized partner together with a method of second choice like condom, diaphragm, or cup pessary Exclusion Criteria: Criteria regarding gastrointestinal conditions: Short bowel syndrome Ileostomy, colostomy or rectal pouch Relapse during screening Criteria regarding medical history: History of or existence of active tuberculosis History of or existence of urolithiasis History of or existence of human immune deficiency virus (HIV), Hepatitis B or C History of malignancy within the past five years (excluding basal cell carcinoma of the skin) Previous opportunistic infection History of serious drug sensitivity Criteria regarding concomitant diseases: Significant cardiac arrhythmia, bradycardia or tachycardia or any other significant finding in the electrocardiogram (ECG) Congestive heart failure Uncontrolled arterial hypertension Uncontrolled asthma Renal disease Renal insufficiency defined as glomerular filtration rate (GRF) <50 ml/min/1.73 m² (estimated GRF according to Cockcroft-Gault) Psychiatric illness Known or suspected immunodeficiency Laboratory abnormalities: hemoglobin <8.5 g/dl, white blood cell count <3500/mm³, platelet count <125 000/mm³, clinically relevant elevation of liver enzymes, serum creatinine level >=1.4 mg/dl, hematuria (>=10 erythrocytes/field on dipsticks) Criteria regarding concomitant circumstances: Pregnancy, lactation History of alcohol and/or drug dependence Heavy smoking (more than 20 cigarettes per day) Use of prohibited drugs or treatments Patient not able or not willing to follow study procedures due to physical or psychological limitations or language problems Participation in another investigational drug or vaccine trial within the last three months or concurrently with this study Vaccination with life attenuated viruses within 4 weeks prior to study start Patient with any medical condition which, in the opinion of the investigator or his designee, could jeopardize or compromise the ability of the patient to participate in the trial Patients possibly dependent on the investigator or the sponsor

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 70 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

| Intervention/Treatment |
| --- |
|Drug: SC12267 (4SC-101)|Peroral administration of SC12267 (4SC-101) in patients with Inflammatory Bowel Disease over 12 weeks.|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of SC12267 (4SC-101) at a dose of 35 mg once daily in patients with Crohn's Disease (CD) or Ulcerative Colitis (UC) after a 12 week therapy as measured by the number of patients with complete or partial response. |  | after a 12 week therapy | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The secondary objective of this study is to evaluate the safety and tolerability of SC12267 (4SC-101) at a dose of 35 mg once daily in patients with CD or UC and to explore plasma levels (trough values) of SC12267 (4SC-101). |  | after a 12 week therapy | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Inflammatory Bowel Disease (IBD), Crohn's Disease, Ulcerative Colitis, SC12267, 4SC-101, Phase II