A4250, an IBAT Inhibitor in Pediatric Cholestasis

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-08-31

Study Completion Date

2017-03-17

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384

NCT02963077

Orphan Cholestatic Liver Diseases Primary Biliary Cirrhosis Progressive Familial Intrahepatic Cholestasis +1 more

COMPLETED PHASE1

Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

NCT01903460

Alagille Syndrome

COMPLETED PHASE2

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT02057692

Alagille Syndrome

COMPLETED PHASE2

An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects

NCT03082937

Orphan Cholestatic Liver Diseases Progressive Familial Intrahepatic Cholestasis Alagille Syndrome +1 more

COMPLETED PHASE1

Safety and Efficacy Study of Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis

NCT01088607

Primary Sclerosing Cholangitis

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pediatric Cholestasis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

A4250 1

Dose I

Group Type ACTIVE_COMPARATOR

A4250

Intervention Type DRUG

A4250

A4250 2

Dose 2

Group Type ACTIVE_COMPARATOR

A4250

Intervention Type DRUG

A4250

A4250 3

Dose 3

Group Type ACTIVE_COMPARATOR

A4250

Intervention Type DRUG

A4250

A4250 4

Dose 4

Group Type ACTIVE_COMPARATOR

A4250

Intervention Type DRUG

A4250

A4250 5

Dose 5

Group Type ACTIVE_COMPARATOR

A4250

Intervention Type DRUG

A4250

A4250 6

Dose 6

Group Type ACTIVE_COMPARATOR

A4250

Intervention Type DRUG

A4250

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

A4250

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

1 Year

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No