Oncology Cardiology / Cardiovascular Respiratory / COPD / Asthma Infectious Disease Neurology Diabetes / Metabolic Rheumatology Gastroenterology Mental Health / Psychiatry Women's Health Dermatology Men's Health Rare Diseases
Clinical Trial ● Currently Recruiting Phase III NCT05035030

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

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome — Recruiting • Phase III • Cardiology / Cardiovascular • NCT05035030.

📅 01 Apr 2026 ⏱ 2 min read
Currently Recruiting
This trial is actively seeking participants in the UK. Discuss eligibility with your patient before referring.
Status
Currently Recruiting
Phase
Phase III
NCT ID
NCT05035030
Sponsor
Albireo, an Ipsen Company
Start
2021-09-03
ClinicaliQ Trial Snapshot
  • Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome — Recruiting • Phase III • Cardiology / Cardiovascular • NCT05035030.
  • 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….
  • Sponsor: Albireo, an Ipsen Company.

Verify eligibility, endpoints and current status on the original source registry before acting on this summary.

Use This Page For
  • Quick orientation before opening the registry record.
  • Checking recruitment status, phase and sponsor at a glance.
  • Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying

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).…

Eligibility Snapshot
  • : 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.

Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.

Full Trial Details
View this trial on the source registry
Eligibility criteria, protocol, and results when available
View Trial ↗
Share: Twitter/X LinkedIn
Related

Related Clinical Intelligence

Guidelines, Drug Science, safety briefs and education connected to this trial area.

Clinical Brief
Weight-loss jabs will be offered on NHS for people at risk of further heart attacks
Cardiology / Cardiovascular · BBC Health · 31 Mar 2026
GLP-1 receptor agonists (weight-loss jabs) will now be offered on NHS to over 1 million people in England at high cardiovascular risk,…
View brief →
Guideline
2023 ESC Guidelines for the Management of Acute Coronary Syndromes
Cardiology / Cardiovascular · 30 Mar 2026
Covers rapid diagnosis and risk stratification for acute coronary syndromes (ACS) including STEMI and NSTEMI • Provides evidence-based recommendations for reperfusion strategies…
View guideline →
Guideline
Surgical insertion of a catheter-based left ventricular microaxial flow pump for cardiogenic shock
Cardiology / Cardiovascular · 25 Mar 2026
Insert a catheter-based left ventricular microaxial flow pump in patients with acute cardiogenic shock refractory to medical therapy and inotropic support, as…
View guideline →
Guideline
Heart Failure: Diagnosis and Management (NICE NG106)
Cardiology / Cardiovascular · 27 Mar 2026
Use NT-BNP or BNP testing in primary care to rule out heart failure; refer urgently to secondary care if elevated with compatible…
View guideline →
Guideline
Percutaneous insertion of a catheter-based left ventricular microaxial flow pump for cardiogenic shock
Cardiology / Cardiovascular · 25 Mar 2026
Percutaneous insertion of catheter-based left ventricular microaxial flow pumps may be considered for patients with acute cardiogenic shock refractory to medical therapy…
View guideline →
Guideline
2023 ESC Guidelines on the Management of Endocarditis
Cardiology / Cardiovascular · 30 Mar 2026
Covers diagnostic criteria for infective endocarditis (Duke criteria) and appropriate imaging modalities • Provides antimicrobial treatment recommendations based on causative organisms and…
View guideline →