Double-blind, randomized, placebo-controlled, phase III study comparing norursodeoxycholic acid capsules with placebo in the treatment of primary sclerosing cholangitis

Phase 3

Recruiting

• Conditions: Primary Sclerosing Cholangitis (PSC); 10019654

• Registration Number: NL-OMON54551
• Lead Sponsor: Dr. Falk Pharma GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 18 June 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 15

Inclusion Criteria

1. Signed Informed Consent
2. Males or Females
3. Verified PSC
4. Liver Biopsy available
8. Women of child-bearing potential who are sexually active have to apply a
highly effective method of brith control

For open-label extension (OLE) phase:
1. Signed Informed Consent
2. DBE phase completed with Visit 22

Exclusion Criteria

1. History or presence of other concomitant liver diseases
4. Secondary causes of Sclerosing Cholangitis
11. Total bilirubin > 4.0 mg/dl (>68 µmol/L) at screening or baseline
13. Any relevant infectious disease (e.g. AIDS)
14. Abnormal renal function
15. Thyroid-stimulating hormone (TSH) > ULN at screening (elevated levels
[4.2-10 µU/mL] are acceptable if fT4 is measured and within the normal range)
17. Any active malignant disease
18. Known intolerance/hypersensitivity to study drug, or drugs of similar
chemical structure or pharmacological profile
19. Well-founded doubt about the patient's cooperation
20. Existing or intented pregnancy or breast-feeding
21. Participation in another clinical trial within the last 30 days

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Partial normalization of s-ALP to < 1.5x ULN and no worsening of disease stage<br /><br>as determined by the Ludwig stage at the week 96 visit compared to<br /><br>baseline.<br /><br><br /><br>The primary endpoint consists of two criteria:<br /><br><br /><br>1) s-ALP criterion: yes = if s-ALP < 1.5x ULN at week 96 (partial<br /><br>normalization), no = if s-ALP >= 1.5x ULN at week 96. If no value at week 96<br /><br>exists, the s-ALP criterion is *no*.<br /><br><br /><br>2) Histological criterion: yes = no worsening of the Ludwig stage; no =<br /><br>otherwise or if no Ludwig stage is available at week 96.<br /><br><br /><br>The primary endpoint is:<br /><br>- Yes = if both criteria above are *yes*.<br /><br>- No = otherwise.</p><br>