A4250, an IBAT Inhibitor in Pediatric Cholestasis

Phase 2

Completed

• Conditions: Pediatric Cholestasis
• Interventions: Drug: A4250

• Registration Number: NCT02630875
• Lead Sponsor: Albireo

Brief Summary

This study will evaluate A4250 (IBATinhibitor) as a treatment option in pediatric patients with chronic cholestasis with main emphasis on safety evaluation and on effects on pruritus

Detailed Description

The primary aims of this Phase II exploratory study in patients treated with A4250 due to cholestasis induced pruritus are to:

* Assess the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent SAEs

* Explore changes in serum total bile acids after a four week treatment period

Secondary safety aims include assessment of the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent AEs and changes in safety parameters including laboratory tests and vital signs

Secondary efficacy aims are to:

* Demonstrate the efficacy of A4250, orally administered during a four week treatment period, on liver biochemistry variables and on pruritus parameters

* Evaluate the pharmacokinetic properties of A4250 orally administered first as a single dose and then after a four week treatment period

* Evaluate changes in VAS-itching score after a four week treatment period

Study Timeline

• Study Start: 2015-08
• Study First Submitted: 2015-11-23
• Study First Submitted QC: 2015-12-14
• Study First Posted: 2015-12-15
• Primary Completion: 2017-03-17
• Study Completion: 2017-03-17
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-06
• Last Update Posted: 2024-03-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

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

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A4250 2	A4250	Dose 2
A4250 1	A4250	Dose I
A4250 3	A4250	Dose 3
A4250 4	A4250	Dose 4
A4250 5	A4250	Dose 5
A4250 6	A4250	Dose 6

Primary Outcome Measures

Name	Time	Method
AE evaluation	4 wks	Treatment-emergent SAEs Adverse events

Secondary Outcome Measures

Name	Time	Method
Bile acid changes	4 weeks	Evaluation of bile acids

Trial Locations

Locations (7)

Gastroenterology/Hepatology, University Hospital for Children and Adolescents; 🇩🇪 Tuebingen, Germany

Henrik Arnell; 🇸🇪 Stockholm, Sweden

King's College Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom

Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital; 🇫🇷 Paris, France

Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover; 🇩🇪 Hannover, Germany

Department of Pediatric and Adolescent Medicine Rigshospitalet; 🇩🇰 Copenhagen, Denmark

Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud; 🇫🇷 Paris, Orsay, France