Clinical trial NCT02630875: A4250, an IBAT Inhibitor in Pediatric Cholestasis
Official title: An Exploratory Phase II Study to Demonstrate the Safety and Efficacy of A4250 in Children With Cholestatic Pruritus
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pediatric Cholestasis
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2015-12-15
Enrollment: 24
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Eligibility 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

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

Primary outcomes:
measure: AE evaluation
timeFrame: 4 wks
Secondary outcomes:
measure: Bile acid changes
timeFrame: 4 weeks
Other outcomes:
Locations:
city: Copenhagen
country: Denmark
facility: Department of Pediatric and Adolescent Medicine Rigshospitalet
city: Paris
country: France
facility: Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud
state: Orsay
city: Paris
country: France
facility: Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital
city: Hannover
country: Germany
facility: Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover
city: Tuebingen
country: Germany
facility: Gastroenterology/Hepatology, University Hospital for Children and Adolescents
city: Stockholm
country: Sweden
facility: Henrik Arnell
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: