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Ursodeoxycholic Acid Plus Budesonide Versus Ursodeoxycholic Acid Alone in Primary Biliary Cirrhosis (PBC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00746486
Recruitment Status : Terminated (Study stopped based on the recommendation of the IDMC after a planned Interim Analysis)
First Posted : September 4, 2008
Last Update Posted : January 28, 2020
Information provided by (Responsible Party):
Dr. Falk Pharma GmbH

Brief Summary:
The study is aimed to compare the efficacy and tolerability of a combination therapy with ursodeoxycholic acid (12-16 mg/kg body weight (BW)/d) plus budesonide (9 mg/d) vs. ursodeoxycholic acid (12-16 mg/kg BW/d) plus placebo in the treatment of PBC. Depending on ALT values 6 mg/d budesonide are allowed. The study population will be patients with PBC at risk for disease progression. It is assumed that the combination therapy will result in a decrease of treatment failures after 3 years of treatment.

Condition or disease Intervention/treatment Phase
Primary Biliary Cirrhosis Drug: budesonide Drug: budesonide placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 62 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-blind, Randomised, Placebo-controlled, Multi-centre Phase III Clinical Study Comparing the Combination of Ursodeoxycholic Acid Capsules Plus Budesonide Capsules to Ursodeoxycholic Acid Capsules Plus Placebo in the Treatment of Primary Biliary Cirrhosis
Study Start Date : February 2009
Actual Primary Completion Date : October 2019
Actual Study Completion Date : October 2019

Arm Intervention/treatment
Experimental: A
One budesonide 3 mg capsule TD or one budesonide 3 mg capsule BD and 12-16 mg ursodeoxycholic acid/kg BW/d
Drug: budesonide
One budesonide 3 mg capsule TD or one budesonide 3 mg capsule BD and 12-16 mg ursodeoxycholic acid/kg BW/d for 3 years

Active Comparator: B
One placebo capsule TD or One placebo capsule BD and 12-16 mg ursodeoxycholic acid/kg BW/d
Drug: budesonide placebo
One placebo capsule TD or One placebo capsule BD and 12-16 mg ursodeoxycholic acid/kg BW/d for 3 years

Primary Outcome Measures :
  1. Rate of patients without treatment failure after 3 years of treatment [ Time Frame: 3 years, LOCF ]

Secondary Outcome Measures :
  1. course of pruritus [ Time Frame: 3 years, LOCF ]
  2. course of fatigue [ Time Frame: 3 years, LOCF ]
  3. course of Mayo Risk score [ Time Frame: 3 years, LOCF ]
  4. bone mineral density [ Time Frame: 3 years, LOCF ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Signed informed consent
  2. Age ≥ 18 years
  3. UDCA treatment for at least 6 months prior to inclusion
  4. Liver biopsy compatible with PBC
  5. Liver biopsy performed within the last 6 months prior to inclusion
  6. PBC patients at risk of disease progression based on one or more of the following criteria:

    • Serum alkaline phosphatase ≥ 3 times the upper limit of normal at any time since diagnosis of PBC and ALT ≥ 2 times upper limit of normal or
    • Total Bilirubin ≥ 1.0 mg/dl (≥ 17 µmol/L) or
    • Moderate to severe periportal or periseptal lymphocytic interface hepatitis or
    • Periportal and portal fibrosis with numerous septa (Ludwig stage III) without cirrhosis
  7. Type 2 anti-mitochondrial antibodies > 1:40 by direct immunofluorescence
  8. Women of child-bearing potential have to apply appropriate contraceptive methods, e.g., hormonal contraception, intrauterine device (IUD), double-barrier method of contraception (e.g., use of a condom and spermicide), partner has undergone vasectomy and subject is in monogamous relationship. The investigator is responsible for determining whether the subject has adequate birth control for study participation

Exclusion Criteria:

  1. Histologically proven cirrhosis
  2. Positive Hepatitis B or C serology
  3. Positive HIV serology
  4. Primary Sclerosing Cholangitis
  5. Wilson's-Disease
  6. Celiac Disease (blood tests and/or oesophago-gastro-duodenoscopy with histological examination to be performed)
  7. α1-anti-Trypsin-deficiency
  8. Haemochromatosis
  9. Autoimmune-Hepatitis (AIH; defined by an Alvarez score > 15 without treatment or ≥ 17 with treatment); Note: PBC/AIH overlap disease, treated insufficiently with UDCA monotherapy may be enrolled
  10. Treatment with any of the following drugs within the last 3 months prior to inclusion: colchicine, corticosteroids, azathioprine or other immunosuppressive drugs (e.g. cyclosporine, methotrexate), chlorambucil, D-penicillamine, fibrates, or antihyperlipidemic drugs
  11. Treatment with ketoconazole or other CYP3A inhibitors within the last 4 weeks before baseline; rifampicin (up to 600 mg/d) is allowed to treat pruritus until baseline
  12. Sonographic or endoscopic signs of portal hypertension
  13. Ascites or history of ascites
  14. Hepatic encephalopathy or history of hepatic encephalopathy
  15. Total bilirubin > 3.0 mg/dl (> 50 µmol/L)
  16. Albumin < 36 g/L
  17. Prothrombin ratio < 70%
  18. Platelet count < 135.000/mm3
  19. Osteoporosis proven by bone densitometry
  20. Diabetes mellitus, defined as B-Glucose > 125 mg/dl on an empty stomach (even when controlled)
  21. Hypertension, defined as persistent raised blood pressure > 140/90 mmHg
  22. Suspected non-compliance of the patient (suspected difficulties to comply with the study period of 36 months)
  23. Severe co-morbidity substantially reducing life expectancy
  24. Known intolerance/hypersensitivity/resistance to study drugs or drugs of similar chemical structure or pharmacological profile
  25. Existing or intended pregnancy or breast-feeding
  26. Participation in another clinical trial within the last 30 days, simultaneous participation in another clinical trial, or previous participation in this trial

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00746486

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Hôpital Saint-Antoine
Paris, France, 75571
Universitätsklinikum Bonn
Bonn, NRW, Germany, 53105
Sponsors and Collaborators
Dr. Falk Pharma GmbH
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Principal Investigator: Raoul Poupon, Professor Hôpital Saint-Antoine, 75571 Paris, France
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Dr. Falk Pharma GmbH Identifier: NCT00746486    
Other Study ID Numbers: BUC-56/PBC
First Posted: September 4, 2008    Key Record Dates
Last Update Posted: January 28, 2020
Last Verified: January 2020
Additional relevant MeSH terms:
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Liver Cirrhosis
Liver Cirrhosis, Biliary
Pathologic Processes
Liver Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Anti-Inflammatory Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Hormones, Hormone Substitutes, and Hormone Antagonists