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Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (ASSERT-EXT)

Primary Purpose

Alagille Syndrome

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Odevixibat
Sponsored by
Albireo
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alagille Syndrome

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Completion of the 24-week Treatment Period of Study A4250-012
  2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study
  3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study
  4. Sexually active males and females must agree to use a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug.

Exclusion Criteria:

  1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
  2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012
  3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
  4. Known hypersensitivity to any components of odevixibat

Sites / Locations

  • UCSF
  • Johns Hopkins Hospital
  • Boston Children's Hospital
  • Children's Mercy Hospital and Clinics
  • The Childrens Hospital at Montefiore Albert Einstein School of Medicine
  • Cliniques Universitaires Saint-Luc Bruxelles
  • Antenne pediatrique du CIC-Hopital Jeanne De Flandre
  • Hopital Necker Enfants Malades
  • Charité - Universitätsmedizin Berlin
  • Medizinische Hochschul
  • Universitatsklinik fur Kinder-und Jugendmedizin Tubingen
  • AOU Meyer
  • Azienda Ospedale University
  • Ospedale Pediatrico Bambino Gesu
  • University of Malaya Medical Center
  • Universitair Medisch Centrum Groningen
  • University Medical Center
  • Instytut Pomnik-Centrum Zdrowia Dzieck
  • Istanbul University Istanbul Medical Faculty Hospital
  • King's College Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Odevixibat (A4250)

Arm Description

Capsules for oral administration once daily for 72 weeks.

Outcomes

Primary Outcome Measures

Change in pruritus
Change from baseline in scratching score as measured by the Albireo Observer-Reported Outcome Caregiver Instrument

Secondary Outcome Measures

Change in serum bile acids
Change from baseline in serum bile acids
Change in Quality of Life
Change from baseline in quality-of-life assessment as measured by the Pediatric Quality of Life Inventory Questionnaire
Change in Sleep Parameters
Change from baseline in sleep parameters as measured by the Albireo Patient and Observer-Reported Outcome instruments
Change in Global Symptom Relief
Change from baseline in global symptom relief as measured by both the Global Impression of Symptoms and Global Impression of Change questionnaires
Safety and Tolerability
Safety and Tolerability as assessed by the Number of Participants with Treatment-emergent Adverse Events as Assessed by CTCAE v5.0

Full Information

First Posted
August 13, 2021
Last Updated
October 17, 2022
Sponsor
Albireo
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1. Study Identification

Unique Protocol Identification Number
NCT05035030
Brief Title
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Acronym
ASSERT-EXT
Official Title
An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 3, 2021 (Actual)
Primary Completion Date
February 2024 (Anticipated)
Study Completion Date
March 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Albireo

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)
Detailed Description
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
63 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Odevixibat (A4250)
Arm Type
Experimental
Arm Description
Capsules for oral administration once daily for 72 weeks.
Intervention Type
Drug
Intervention Name(s)
Odevixibat
Other Intervention Name(s)
A4250
Intervention Description
Odevixibat is a small molecule and selective inhibitor of IBAT.
Primary Outcome Measure Information:
Title
Change in pruritus
Description
Change from baseline in scratching score as measured by the Albireo Observer-Reported Outcome Caregiver Instrument
Time Frame
Baseline to week 72
Secondary Outcome Measure Information:
Title
Change in serum bile acids
Description
Change from baseline in serum bile acids
Time Frame
Baseline to week 72
Title
Change in Quality of Life
Description
Change from baseline in quality-of-life assessment as measured by the Pediatric Quality of Life Inventory Questionnaire
Time Frame
Baseline to week 72
Title
Change in Sleep Parameters
Description
Change from baseline in sleep parameters as measured by the Albireo Patient and Observer-Reported Outcome instruments
Time Frame
Baseline to week 72
Title
Change in Global Symptom Relief
Description
Change from baseline in global symptom relief as measured by both the Global Impression of Symptoms and Global Impression of Change questionnaires
Time Frame
Baseline to Weeks, 4, 12, 24, 48, and 72
Title
Safety and Tolerability
Description
Safety and Tolerability as assessed by the Number of Participants with Treatment-emergent Adverse Events as Assessed by CTCAE v5.0
Time Frame
Baseline to week 72

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Completion of the 24-week Treatment Period of Study A4250-012 Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study Sexually active males and females must agree to use a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug. Exclusion Criteria: Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy Patients who were not compliant with study drug treatment or procedures in Study A4250-012 Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study Known hypersensitivity to any components of odevixibat
Facility Information:
Facility Name
UCSF
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Mercy Hospital and Clinics
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64018
Country
United States
Facility Name
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Cliniques Universitaires Saint-Luc Bruxelles
City
Bruxelles
ZIP/Postal Code
1200
Country
Belgium
Facility Name
Antenne pediatrique du CIC-Hopital Jeanne De Flandre
City
Lille
Country
France
Facility Name
Hopital Necker Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Charité - Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Medizinische Hochschul
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Facility Name
Universitatsklinik fur Kinder-und Jugendmedizin Tubingen
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
AOU Meyer
City
Florence
Country
Italy
Facility Name
Azienda Ospedale University
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesu
City
Rome
ZIP/Postal Code
00165
Country
Italy
Facility Name
University of Malaya Medical Center
City
Kuala Lumpur
ZIP/Postal Code
59100
Country
Malaysia
Facility Name
Universitair Medisch Centrum Groningen
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
University Medical Center
City
Utrecht
Country
Netherlands
Facility Name
Instytut Pomnik-Centrum Zdrowia Dzieck
City
Warszawa
ZIP/Postal Code
04-730
Country
Poland
Facility Name
Istanbul University Istanbul Medical Faculty Hospital
City
Istanbul
Country
Turkey
Facility Name
King's College Hospital
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

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