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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05035030
Recruitment Status : Enrolling by invitation
First Posted : September 5, 2021
Last Update Posted : March 10, 2022
Sponsor:
Information provided by (Responsible Party):
Albireo

Brief Summary:
An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

Condition or disease Intervention/treatment Phase
Alagille Syndrome Drug: Odevixibat Phase 3

Detailed Description:
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)
Actual Study Start Date : September 3, 2021
Estimated Primary Completion Date : October 2023
Estimated Study Completion Date : October 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 72 weeks.
Drug: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Other Name: A4250




Primary Outcome Measures :
  1. Change in pruritus [ Time Frame: Baseline to week 72 ]
    Change from baseline in scratching score as measured by the Albireo Observer-Reported Outcome Caregiver Instrument


Secondary Outcome Measures :
  1. Change in serum bile acids [ Time Frame: Baseline to week 72 ]
    Change from baseline in serum bile acids

  2. Change in Quality of Life [ Time Frame: Baseline to week 72 ]
    Change from baseline in quality-of-life assessment as measured by the Pediatric Quality of Life Inventory Questionnaire

  3. Change in Sleep Parameters [ Time Frame: Baseline to week 72 ]
    Change from baseline in sleep parameters as measured by the Albireo Patient and Observer-Reported Outcome instruments

  4. Change in Global Symptom Relief [ Time Frame: Baseline to Weeks, 4, 12, 24, 48, and 72 ]
    Change from baseline in global symptom relief as measured by both the Global Impression of Symptoms and Global Impression of Change questionnaires

  5. Safety and Tolerability [ Time Frame: Baseline to week 72 ]
    Safety and Tolerability as assessed by the Number of Participants with Treatment-emergent Adverse Events as Assessed by CTCAE v5.0



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05035030


Locations
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United States, California
UCSF
San Francisco, California, United States, 94158
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, New York
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
Bronx, New York, United States, 10467
France
Hôpital Femme Mère Enfant de Lyon
Bron, France, 69677
Hopital Necker Enfants Malades
Paris, France, 75015
Poland
Instytut Pomnik-Centrum Zdrowia Dzieck
Warszawa, Poland, 04-730
United Kingdom
King's College Hospital
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Albireo
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Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT05035030    
Other Study ID Numbers: A4250-015
First Posted: September 5, 2021    Key Record Dates
Last Update Posted: March 10, 2022
Last Verified: March 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alagille Syndrome
Syndrome
Disease
Pathologic Processes
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn