Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (NCT05035030) | Clinical Trial Compass
RecruitingPhase 3
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
United States, Australia, Belgium70 participantsStarted 2021-09-03
Plain-language summary
The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS).
The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis).
The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
. Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1
. Body weight ≥2 kg at Study Day 1
. Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required .
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change from baseline in pruritus
Timeframe: Baseline to week 72 (cohort 1).
2
Percentage of participants with Treatment Emergent Adverse Event (TEAEs) and Serious Adverse Events (SAEs)
Timeframe: Baseline to week 12 (cohort 2).
3
Percentage of participants with clinically significant changes from baseline in Physical Examination
Timeframe: Baseline to week 12 (cohort 2).
4
Percentage of participants with clinically significant changes in Laboratory Parameters
Timeframe: Baseline to week 12 (cohort 2).
5
Percentage of participants with clinically significant changes from baseline in Vital Signs.
Timeframe: Baseline to week 12 (cohort 2).
6
Change from baseline in concomitant medications.
Timeframe: Baseline to week 12 (cohort 2).
7
Change from baseline in fat-soluble vitamin levels.
. 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
. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following: