An Exploratory Phase II Study to demonstrate the Safety and Efficacy of A4250 in Children with Cholestatic Pruritus

Phase 1

• Conditions: Patients wih Cholestatic Pruritus; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2015-001157-32-GB
• Lead Sponsor: Albireo AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-08-09
• Study Completion: 2017-04-05
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 24

Inclusion Criteria

The main inclusion criteria for study participation eligibility will be:
• Diagnosis of pruritus due to chronic cholestasis based on history and investigator judgment
o This will include but will not be restricted to patients with Progressive familial
intrahepatic cholestasis (PFIC), Alagille syndrome (ALGS), Biliary Atresia and
Sclerosing Cholangitis
• Laboratory markers of cholestasis identified within 3 months before Visit 1
• Serum total bile acids at least 2 times above upper limit of normal
• A VAS-itch of at least 3 (average of 7 days) on a 10-graded VAS at Visit 2
• The caretaker/patient reports having understood and has signed the ICF and is willing to comply with all study visits and assessments
• The patient is a male or non-pregnant female =12 months of age and <18 years of age with a body weight exceeding 7 kg
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

The main exclusion criteria for study participation eligibility will be:
• Any condition that in the opinion of the investigator constitutes a risk for the patient or a contraindication for participation and completion of the study, or could interfere with study objectives, conduct, or evaluations
• Clinical or biochemical signs of decompensated liver disease (such as ascites)
• Liver transplantation
• Structural abnormality of the GI tract (biliary diversion procedures accepted).
• Known, active, clinically significant acute or chronic infection, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infective treatment within 4 weeks of treatment start (study day 1) or completion of oral anti-infective treatment within 2 weeks prior to start of screening period
• A history of cancer with last date of proven disease activity/presence of malignancy within 5 years, except for adequately treated basal cell carcinoma of the skin, cervical dysplasia, or carcinoma in situ of the skin or the cervix
• Other reason for pruritus than chronic cholestasis such as treatment refractory atopic dermatitis, other primary skin diseases etc.
• Treatment with bile acid sequestrants (cholestyramine, colesevelam, colestipol or similar) during the screening period
• Chronic kidney disease with an impaired renal function and a GFR<70 ml/min1.73 m2
• Active substance abuse in the year before screening
• A history of a psychiatric disorder requiring hospitalization or suicide attempt in the 2 years prior to Screening;
• Participation in any investigational clinical study within 30 days prior to Screening, or plans to participate in another clinical study during this study;
• Ongoing pregnancy, breast-feeding or lactation.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified