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A4250, an IBAT Inhibitor in Pediatric Cholestasis

Primary Purpose

Pediatric Cholestasis

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

A4250

About this trial

This is an interventional treatment trial for Pediatric Cholestasis

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Department of Pediatric and Adolescent Medicine Rigshospitalet
Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud
Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital
Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover
Gastroenterology/Hepatology, University Hospital for Children and Adolescents
Henrik Arnell
King's College Hospital NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Active Comparator

Arm Label

A4250 1

A4250 2

A4250 3

A4250 4

A4250 5

A4250 6

Arm Description

Dose I

Dose 2

Dose 3

Dose 4

Dose 5

Dose 6

Outcomes

Primary Outcome Measures

AE evaluation

Treatment-emergent SAEs Adverse events

Secondary Outcome Measures

Bile acid changes

Evaluation of bile acids

Full Information

NCT ID

NCT02630875

First Posted

November 23, 2015

Last Updated

March 28, 2018

Sponsor

Albireo

1. Study Identification

Unique Protocol Identification Number

NCT02630875

Brief Title

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

2. Study Status

Record Verification Date

March 2018

Overall Recruitment Status

Completed

Study Start Date

August 2015 (undefined)

Primary Completion Date

March 17, 2017 (Actual)

Study Completion Date

March 17, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Albireo

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pediatric Cholestasis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

24 (Actual)

8. Arms, Groups, and Interventions

Arm Title

A4250 1

Arm Type

Active Comparator

Arm Description

Dose I

Arm Title

A4250 2

Arm Type

Active Comparator

Arm Description

Dose 2

Arm Title

A4250 3

Arm Type

Active Comparator

Arm Description

Dose 3

Arm Title

A4250 4

Arm Type

Active Comparator

Arm Description

Dose 4

Arm Title

A4250 5

Arm Type

Active Comparator

Arm Description

Dose 5

Arm Title

A4250 6

Arm Type

Active Comparator

Arm Description

Dose 6

Intervention Type

Drug

Intervention Name(s)

A4250

Intervention Description

A4250

Primary Outcome Measure Information:

Title

AE evaluation

Description

Treatment-emergent SAEs Adverse events

Time Frame

4 wks

Secondary Outcome Measure Information:

Title

Bile acid changes

Description

Evaluation of bile acids

Time Frame

4 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Mats Ekelund, MD

Organizational Affiliation

Albireo

Official's Role

Study Chair

First Name & Middle Initial & Last Name & Degree

Henrik Arnell, MD

Organizational Affiliation

Karolinska

Official's Role

Principal Investigator

Facility Information:

Facility Name

Department of Pediatric and Adolescent Medicine Rigshospitalet

City

Copenhagen

Country

Denmark

Facility Name

Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud

City

Paris

State/Province

Orsay

Country

France

Facility Name

Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital

City

Paris

Country

France

Facility Name

Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover

City

Hannover

Country

Germany

Facility Name

Gastroenterology/Hepatology, University Hospital for Children and Adolescents

City

Tuebingen

Country

Germany

Facility Name

Henrik Arnell

City

Stockholm

ZIP/Postal Code

17176

Country

Sweden

Facility Name

King's College Hospital NHS Foundation Trust

City

London

Country

United Kingdom

12. IPD Sharing Statement

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A4250, an IBAT Inhibitor in Pediatric Cholestasis

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