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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-001157-32-SE

Enrollment

Registered

2015-03-27

Start date

2015-05-13

Completion date

Unknown

Last updated

2017-05-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Patients wih Cholestatic Pruritus

Interventions

Product Code: A4250 Pharmaceutical Form: Capsule INN or Proposed INN: A4250 Other descriptive name: A4250 Concentration unit: mg milligram(s) Concentration type: range Concentration number: 0.07-20

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Diagnosis of pruritus due to chronic cholestasis based on history and investigator judgment - 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 Are the trial subjects under 18? yes Number of subjects for this age range: 23 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: •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 •Liver transplantation •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

Design outcomes

Primary

Measure	Time frame
Main Objective: •Assess the safety and tolerability of A4250, orally administered •Explore changes in serum total bile acids;Secondary Objective: •Assessment of the safety and tolerability of A4250 •Demonstrate the efficacy of A4250, orally administered during a four week treatment period, on liver biochemistry variables and on pruritus parameters ;Primary end point(s): The primary efficacy endpoint is change in total serum bile acids. The primary safety endpoint will be the occurrence of treatment-emergent SAEs during the four treatment weeks. Description and severity of any SAE will also be reported.;Timepoint(s) of evaluation of this end point: The end of the four week treatment.	—

Measure

Time frame

Main Objective: •Assess the safety and tolerability of A4250, orally administered •Explore changes in serum total bile acids;Secondary Objective: •Assessment of the safety and tolerability of A4250 •Demonstrate the efficacy of A4250, orally administered during a four week treatment period, on liver biochemistry variables and on pruritus parameters ;Primary end point(s): The primary efficacy endpoint is change in total serum bile acids. The primary safety endpoint will be the occurrence of treatment-emergent SAEs during the four treatment weeks. Description and severity of any SAE will also be reported.;Timepoint(s) of evaluation of this end point: The end of the four week treatment.

—

Secondary

Measure	Time frame
Secondary end point(s): •VAS-itch •Whitington itching score •PK •Liver biochemistry evaluation •Occurrence of TEAEs during the whole study period. Description and severity of any AE will also be reported.;Timepoint(s) of evaluation of this end point: The end of four week treatment.	—

Countries

Denmark, Germany, Sweden, United Kingdom

Contacts

Public ContactResponsible Medical Officer

Albireo AB

mats.ekelund@albireopharma.com00460703136910

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026