Norursodeoxycholic acid vs. Placebo in PSC

• Research type

  Research Study

• Full title

  Double-blind, randomized, placebo-controlled, phase II dose-finding study comparing different doses of norursodeoxycholic acid capsules with placebo in the treatment of primary sclerosing cholangitis

• IRAS ID

  108593

• Contact name

  Roger Chapman

• Sponsor organisation

  Dr. Falk Pharma GmbH

• Eudract number

  2011-002754-31

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  This study will be used to determine the efficacy of three doses of Norursodeoxycholic acid (norUDCA) versus placebo for the treatment of Primary Sclerosing Cholangitis (PSC), which will assist in determining the doses of norUDCA that have efficacy and should be evaluated further in phase III. At this time there is no proven successful drug treatment for the disease PSC and therefore this is an important study for this patient population. About 160 patients will be recruited from approximately 11 European countries at hospitals with consultants with a specific interest in this disease area. The study will last between 18 and 26 weeks. During this time the patient will have nine study visits. The first visit will be a screening visit at least 14 days (but up to 70 days), before the start of treatment, to check whether the patient is eligible for the study and whether additional diagnostic examinations must be performed, e.g. MRCP or colonoscopy. The second visit will be performed and the treatment with study medication will be started. The duration of treatment will be 12 weeks and during this time the patient will visit the study doctor for control visits every 2 weeks and finally for an end-of-treatment visit. During these visits the patient will have physical examinations, electrocardiograms, ultrasounds and blood tests. The blood tests will assess whether the drug is successful by checking many different components, but especially liver function. The patients will also have to complete questionnaires to assess their general health including fatigue and pruritus (itching) a common symptom of PSC. The 12-week treatment period will be followed by a 4-week period with a follow-up visit at the end.

• REC name

  South Central - Oxford C Research Ethics Committee

• REC reference

  13/SC/0027

• Date of REC Opinion

  27 Mar 2013

• REC opinion

  Further Information Favourable Opinion