A4250, an IBAT Inhibitor in Pediatric Cholestasis

Pediatric Cholestasis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02630875
• Other study ID #: A4250-003
• Status: Completed
• Phase: Phase 2
• Start date: August 2015
• Est. completion date: March 17, 2017

Study information

• Verified date: March 2024
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: March 17, 2017
• Est. primary completion date: March 17, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Cholestasis
• Pediatric Cholestasis

Intervention

Drug:
• A4250
A4250

Locations

Country	Name	City	State
Denmark	Department of Pediatric and Adolescent Medicine Rigshospitalet	Copenhagen
France	Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital	Paris
France	Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud	Paris	Orsay
Germany	Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover	Hannover
Germany	Gastroenterology/Hepatology, University Hospital for Children and Adolescents	Tuebingen
Sweden	Henrik Arnell	Stockholm
United Kingdom	King's College Hospital NHS Foundation Trust	London

Sponsors (1)

Lead Sponsor	Collaborator
Albireo

Countries where clinical trial is conducted

Denmark,  France,  Germany,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	AE evaluation	Treatment-emergent SAEs Adverse events	4 wks
Secondary	Bile acid changes	Evaluation of bile acids	4 weeks