MedPath

A 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-DE
Lead Sponsor
Albireo AB
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
24
Inclusion Criteria

The inclusion criteria for study participation eligibility are:
• Diagnosis of pruritus due to chronic cholestasis based on history and Investigator judgment
-> This will include but will not be restricted to patients with PFIC, ALGS, Biliary Atresia and Sclerosing Cholangitis
• Laboratory markers of cholestasis identified within 3 months of Visit 1
• Serum total bile acids at least 2 times above upper limit of normal
• The patient has a VAS-itch of at least 3 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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The exclusion criteria for study participation eligibility are:
• 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 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 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, with the exception of the low dose of this 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
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
© Copyright 2025. All Rights Reserved by MedPath