Ongoing Clinical Trials for Alagille Syndrome
Currently, there are 2 ongoing clinical trials investigating treatments for Alagille syndrome, a rare genetic disorder affecting the liver and other organs. These trials are studying medications that target bile acid management to help reduce symptoms such as severe itching and liver damage. The trials are taking place across several European countries, including France, Italy, Spain, Germany, Netherlands, Belgium, and Poland.
Clinical trial locations
- Belgium
- France
- Germany
- Italy
- Netherlands
- Poland
- Spain
Study on Long-Term Safety and Effects of Maralixibat for Patients with Alagille Syndrome
This trial is investigating Livmarli, an oral solution containing maralixibat, for the long-term treatment of Alagille syndrome. The study will continue until 2035, providing an extensive look at how well patients tolerate this medication and its effectiveness in managing symptoms over many years.
Who can join this study:
- Children and adolescents aged 2 to 18 years with a confirmed diagnosis of Alagille syndrome, either through clinical evaluation or genetic testing
- Patients experiencing itching due to chronic cholestasis, which is a long-term condition where bile flow from the liver is reduced or blocked
- Those who are currently prescribed Livmarli or have been prescribed it in the past
- Participants and their caregivers must provide informed consent and, if applicable, assent
- Both males and females are eligible
Who cannot join this study:
- Patients without Alagille syndrome
- Children younger than 2 years old or older than 18 years
- Those not meeting the specific clinical trial group requirements
What the study involves:
The trial focuses on evaluating how well patients tolerate Livmarli over an extended period and monitoring for any side effects, particularly liver-related issues. Researchers will observe changes in liver function, the severity of itching, levels of bile acids in the blood, and the use of other medications for liver disease. The study will also assess how well patients tolerate the starting dose and any changes in dosage, with adjustments made if necessary based on tolerability.
Investigational drug:
Livmarli works as a bile acid modulator at the molecular level, targeting specific pathways in the liver to help reduce bile acid buildup. This can help alleviate symptoms and improve liver function in patients with Alagille syndrome.
Long-term Study on the Safety and Effectiveness of Odevixibat for Patients with Alagille Syndrome
This clinical trial is examining odevixibat, also known by its code name A4250, for the long-term management of Alagille syndrome. The study is designed to last up to 72 weeks and will conclude by April 2027.
Who can join this study:
The study includes two different groups of participants:
Cohort 1:
- Patients who have already completed a previous 24-week study of odevixibat (Study A4250-012)
- Willing to sign an informed consent form (participants who turn 18 during the study must sign again)
- Able to use an electronic diary device with their caregiver
- Sexually active participants must agree to use reliable birth control methods from consent through 90 days after the last dose
Cohort 2:
- Infants 11 months old or younger with clinically confirmed Alagille syndrome
- Weighing at least 2 kg at the start of the study
- Born at a gestational age of at least 36 weeks, or if born between 32 and 36 weeks, must have a postmenstrual age of at least 36 weeks
- Parent or legal guardian must provide informed consent
Who cannot join this study:
- Patients without Alagille syndrome
- Those who have not completed the previous A4250-012 study (for Cohort 1)
- Patients unable to follow study procedures or take the medication as required
- Those with other medical conditions that might interfere with the study or pose additional risks
What the study involves:
Participants will take odevixibat in capsule form by mouth. Throughout the study, researchers will monitor changes in symptoms such as itching and scratching using caregiver-reported assessments. Additional evaluations include measuring serum bile acid levels, sleep patterns, and quality of life scores at various intervals including Weeks 4, 12, 24, 48, and 72. For Cohort 2, the focus is on evaluating safety and how well young infants tolerate the medication.
Investigational drug:
Odevixibat is classified as a bile acid transporter inhibitor. It works by inhibiting the ileal bile acid transporter, which reduces the reabsorption of bile acids in the intestines. This mechanism helps alleviate severe itching and other symptoms associated with Alagille syndrome by reducing bile acid buildup in the body.
Summary
Both ongoing clinical trials for Alagille syndrome are investigating medications that target bile acid management, representing an important therapeutic approach for this rare genetic disorder. The trials are concentrated in Western European countries, with France and Italy participating in both studies, while Germany, Netherlands, Belgium, and Poland are involved in the odevixibat trial, and Spain is participating in the maralixibat trial.
A notable distinction between the two trials is their scope: the maralixibat study extends until 2035, offering an exceptionally long follow-up period for patients aged 2 to 18 years, while the odevixibat trial runs up to 72 weeks and includes a cohort specifically designed for infants under 12 months old. Both medications work through similar mechanisms as bile acid modulators or inhibitors, aiming to reduce the buildup of bile acids that causes severe itching and liver damage in patients with this condition.
These trials reflect the ongoing research efforts to develop effective long-term treatments for Alagille syndrome, with particular attention to safety monitoring and symptom management across different age groups.
