A4250, an IBAT Inhibitor in Pediatric Cholestasis

• Sponsor: Albireo (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02630875
• Collaborator: (none)
• Enrollment: 24
• Locations: 7
• Arms: 6
• Duration (Months): 19.5
• Patients Per Site: 3.4
• Patients Per Site Per Month: 0.2

Study Details

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

Condition or Disease	Intervention/Treatment	Phase
Pediatric Cholestasis	Drug: A4250	Phase 2

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

Study Design

• Study Type: Interventional
• Actual Enrollment: 24 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Exploratory Phase II Study to Demonstrate the Safety and Efficacy of A4250 in Children With Cholestatic Pruritus
• Study Start Date: Aug 1, 2015
• Actual Primary Completion Date: Mar 17, 2017
• Actual Study Completion Date: Mar 17, 2017

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: A4250 1 Dose I	Drug: A4250; A4250
Active Comparator: A4250 2 Dose 2	Drug: A4250; A4250
Active Comparator: A4250 3 Dose 3	Drug: A4250; A4250
Active Comparator: A4250 4 Dose 4	Drug: A4250; A4250
Active Comparator: A4250 5 Dose 5	Drug: A4250; A4250
Active Comparator: A4250 6 Dose 6	Drug: A4250; A4250

Outcome Measures

Primary Outcome Measures

1. AE evaluation [4 wks]

   Treatment-emergent SAEs Adverse events

Secondary Outcome Measures

1. Bile acid changes [4 weeks]

   Evaluation of bile acids

Eligibility Criteria

Criteria

• Ages Eligible for Study: 1 Year to 18 Years
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Department of Pediatric and Adolescent Medicine Rigshospitalet	Copenhagen		Denmark
2	Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud	Paris	Orsay	France
3	Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital	Paris		France
4	Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover	Hannover		Germany
5	Gastroenterology/Hepatology, University Hospital for Children and Adolescents	Tuebingen		Germany
6	Henrik Arnell	Stockholm		Sweden	17176
7	King's College Hospital NHS Foundation Trust	London		United Kingdom

Sponsors and Collaborators

• Albireo

Investigators

• Study Chair: Mats Ekelund, MD, Albireo
• Principal Investigator: Henrik Arnell, MD, Karolinska

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Albireo
• ClinicalTrials.gov Identifier: NCT02630875
• Other Study ID Numbers: A4250-003
• First Posted: Dec 15, 2015
• Last Update Posted: Mar 29, 2018
• Last Verified: Mar 1, 2018

• Additional relevant MeSH terms: Cholestasis

Study Results

No Results Posted as of Mar 29, 2018