NCT05035030 Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
| NCT ID | NCT05035030 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Albireo, an Ipsen Company |
| Condition | Alagille Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 70 participants |
| Start Date | 2021-09-03 |
| Primary Completion | 2026-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 70 participants in total. It began in 2021-09-03 with a primary completion date of 2026-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: Cohort 1 : 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. Cohort 2 : 1. Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1 2. Body weight ≥2 kg at Study Day 1 3. Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required . 4. Signed parent/legal guardian informed consent. Exclusion Criteria: Cohort 1 : 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 Cohort 2 : 1. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following: 1. Biliary atresia of any kind 2. Progressive familial intrahepatic cholestasis (PFIC) 3. Benign recurrent intrahepatic cholestasis 2. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease 3. Patient with past medical history or ongoing chronic diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae 4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection 5. Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of Study Day 1 or completion of oral anti-infective treatment within 2 weeks prior to the Screening Visit 6. Cancer diagnosis (except for basal cell carcinoma) 7. Chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m2 8. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to the Screening Visit 9. Patient has had a liver transplant, or a liver transplant is planned within 6 months of Study Day 1 10. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 11. International normalized ratio (INR) \>1.4 (the patient may be treated with Vitamin K, and if INR is ≤1.4 at resampling the patient may be enrolled) 12. Serum alanine aminotransferase (ALT) \>10 × upper limit of normal (ULN) at Screening 13. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation 14. Total bilirubin \>15 × ULN at Screening 15. Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases. 16. Patient exposed to alcohol or substance abuse in utero 17. Bile acid or lipid binding resins and medications that slow gastrointestinal motility 18. Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to the Screening Visit, or 5 half-lives of the study agent, whichever is longer 19. 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
Contact & Investigator
Ipsen Medical Director
STUDY DIRECTOR
Ipsen
Frequently Asked Questions
Who can join the NCT05035030 clinical trial?
This trial is open to participants of all sexes, studying Alagille Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05035030 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 70 participants.
Is NCT05035030 currently recruiting?
Yes, NCT05035030 is actively recruiting participants. Contact the research team at clinical.trials@ipsen.com for enrollment information.
Where is the NCT05035030 trial being conducted?
This trial is being conducted at San Diego, United States, San Francisco, United States, Atlanta, United States, Indianapolis, United States and 11 additional locations.
Who is sponsoring the NCT05035030 clinical trial?
NCT05035030 is sponsored by Albireo, an Ipsen Company. The principal investigator is Ipsen Medical Director at Ipsen. The trial plans to enroll 70 participants.