Pediatric Cholestasis
Conditions
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
Interventions
DRUGA4250
A4250
Sponsors
Albireo
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| AE evaluation | 4 wks | Treatment-emergent SAEs Adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Bile acid changes | 4 weeks | Evaluation of bile acids |
Countries
Denmark, France, Germany, Sweden, United Kingdom
Outcome results
None listed