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

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.


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

  1. unable to taper steroids completely within 3 months of starting steroids, without recurrent active disease, or
  2. 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

  1. unable to taper steroids completely within 3 months of starting steroids, without recurrent active disease, or
  2. 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:

  1. 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
  2. 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