NCT02630875

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

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2015

Geographic Reach
5 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2015

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

November 23, 2015

Completed
22 days until next milestone

First Posted

Study publicly available on registry

December 15, 2015

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 17, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 17, 2017

Completed
Last Updated

March 7, 2024

Status Verified

March 1, 2024

Enrollment Period

1.6 years

First QC Date

November 23, 2015

Last Update Submit

March 6, 2024

Conditions

Pediatric Cholestasis

Outcome Measures

Primary Outcomes (1)

  • AE evaluation

    Treatment-emergent SAEs Adverse events

    4 wks

Secondary Outcomes (1)

  • Bile acid changes

    4 weeks

Study Arms (6)

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

A4250DRUG

A4250

A4250 1A4250 2A4250 3A4250 4A4250 5A4250 6

Eligibility Criteria

Age1 Year - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Department of Pediatric and Adolescent Medicine Rigshospitalet

Copenhagen, Denmark

Location

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

Paris, Orsay, France

Location

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

Paris, France

Location

Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover

Hanover, Germany

Location

Gastroenterology/Hepatology, University Hospital for Children and Adolescents

Tübingen, Germany

Location

Henrik Arnell

Stockholm, 17176, Sweden

Location

King's College Hospital NHS Foundation Trust

London, United Kingdom

Location

Study Officials

  • Ipsen Medical Director

    Ipsen

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 23, 2015

First Posted

December 15, 2015

Study Start

August 1, 2015

Primary Completion

March 17, 2017

Study Completion

March 17, 2017

Last Updated

March 7, 2024

Record last verified: 2024-03

Data Sharing

IPD Sharing
Will share

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.

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/).
More information

Locations

SE(1)FR(2)GB(1)DE(2)DK(1)