A4250, an IBAT Inhibitor in Pediatric Cholestasis | NCT02630875

Studies
A4250, an IBAT Inhibitor in Pediatric Cholestasis

NCT02630875

Sponsor

Albireo

Status

Completed

Last Update Posted

Mar 7, 2024Actual

Enrollment

24Actual

Phase

Phase 2

Conditions

Pediatric Cholestasis

Interventions

A4250

Countries

Denmark
France
Germany
Sweden
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2024

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 2015

Primary Completion Date

Mar 17, 2017Actual

Completion Date

Mar 17, 2017Actual

First Submitted Date

Nov 23, 2015

First Submission Date that Met QC Criteria

Dec 14, 2015

First Posted Date

Dec 15, 2015Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 6, 2024

Last Update Posted Date

Mar 7, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

24Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
A4250 1	Active Comparator	Dose I	Drug: A4250
A4250 2	Active Comparator	Dose 2	Drug: A4250
A4250 3	Active Comparator	Dose 3	Drug: A4250
A4250 4	Active Comparator	Dose 4	Drug: A4250
A4250 5	Active Comparator	Dose 5	Drug: A4250
A4250 6	Active Comparator	Dose 6	Drug: A4250

Interventions

Name	Type	Description	Arm Group Labels	Other Names
A4250	Drug	A4250	A4250 1 A4250 2 A4250 3 A4250 4 A4250 5 A4250 6

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
AE evaluation	Treatment-emergent SAEs Adverse events	4 wks

Secondary Outcomes

Measure	Description	Time Frame
Bile acid changes	Evaluation of bile acids	4 weeks

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Year

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Ipsen Medical Director	Ipsen	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Department of Pediatric and Adolescent Medicine Rigshospitalet		Copenhagen			Denmark
Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud

IPD Sharing Statement Module

Plan to Share IPD

Yes

Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.

Types

Not provided

Time Frame

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.

Access Criteria

Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).

URL

https://vivli.org/members/ourmembers/