Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
This study is checking the long-term safety and how well a medicine called odevixibat works for people with Alagille syndrome (ALGS). ALGS is a rare genetic condition that can affect the liver and other body parts, causing problems like yellow skin (jaundice) and severe itching. Odevixibat is already approved in the US for itching in ALGS patients over 12 months old. The study involves two groups: one who has been in a previous study and one with babies under 12 months who have ALGS. Participants will take odevixibat daily and have regular check-ups. The aim is to see if this medicine can help manage symptoms and improve the lives of those with Alagille syndrome over a long period.
במבט חטוף
על מה המחקר הזה?
This research study is focused on a medicine called odevixibat for people who have Alagille syndrome. Alagille syndrome is a rare condition that affects different parts of the body, especially the liver. People with Alagille syndrome can experience symptoms like yellow skin, poor growth, and very uncomfortable itching, which often start when they are very young. Odevixibat has already been approved in the United States to help with the severe itching that babies with Alagille syndrome over 12 months old can experience.
The main goal of this study is to learn more about how safe odevixibat is when taken for a long time, and how well it continues to help with symptoms in people with Alagille syndrome. By studying it over a longer period, doctors hope to understand its full potential and ensure it's a suitable long-term treatment option.
This study is important because it could provide more evidence for a medicine that helps manage some of the challenging symptoms of Alagille syndrome, particularly the severe itching. Better understanding of its long-term use means better care for those living with this condition.
נקודות עיקריות
- The study explores the long-term safety and effectiveness of odevixibat for Alagille syndrome.
- It aims to reduce severe itching and improve quality of life for patients.
- Participants will take the medicine daily and have regular check-ups.
- Two groups are involved: previous study participants and babies under 12 months with Alagille syndrome.
- There's an optional extension to continue treatment if it's beneficial.
- אתה יכול להפסיק להשתתף בכל עת.
מי עשוי להיות זכאי?
This study is looking for two main groups of people. One group includes patients who have already taken part in a previous study (Study A4250-012) on odevixibat and meet certain requirements to continue. The other group is babies with Alagille syndrome who are under 12 months old and weigh at least 2kg.
For those who were in the previous study, you'll need to be willing to use an electronic diary for reporting information. If you're sexually active, both men and women will need to use effective contraception during the study and for 90 days after finishing the medicine.
There are also reasons why someone might not be able to join. For example, if you have severe liver disease (like bleeding from veins in the food pipe, called variceal hemorrhage, or problems with brain function due to liver disease), or if you had issues following instructions in the previous study, you might not be eligible. Babies with other types of liver disease, like biliary atresia, or those who are very unwell, won't be able to participate. Essentially, the doctors need to make sure it's safe for you to take part.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- Do you have a confirmed diagnosis of Alagille syndrome?
- If you're in the baby group, is the baby under 12 months old and at least 2kg?
- If you were in Study A4250-012, did you complete the 24-week treatment period?
- Are you able and willing to attend regular clinic visits?
- Are you willing to use effective contraception if sexually active (for Cohort 1)?
- Do you have any severe liver complications or other serious health issues?
מה כרוך בהשתתפות?
If you join this study, your involvement will depend on which group you are in. If you were in a previous study (Cohort 1), you'll take the medicine odevixibat once a day for about 72 weeks (about 1 year and 5 months). You'll visit the clinic every 4 to 12 weeks during this time. If you're a baby (Cohort 2), you'll take odevixibat daily for 12 weeks, with clinic visits every 4 to 12 weeks.
After this initial treatment period, if you're doing well and want to continue, you might be able to stay on the medicine until it's more widely available. During this 'optional extension period', visits would be less frequent, about every 16 weeks. If you don't continue with the extension, there will be a final safety check-up, either 4 weeks (Cohort 1) or 2 weeks (Cohort 2) after your last dose.
Throughout the study, you'll have regular checks such as blood tests, physical exams, and doctors will assess your overall health. Some patients in Cohort 1 will also collect urine samples and fill out electronic diaries and questionnaires. It's important to keep track of any other medicines you are taking.
סיכונים ותועלות פוטנציאליים
מיקומים (35)
- Rady Children's Hospitalמיקוד מאומתסן דייגו, ארצות הברית
- UCSFמיקוד מאומתסן פרנסיסקו, ארצות הברית
- Children's Healthcare of Atlantaמיקוד מאומתאטלנטה, ארצות הברית
- Riley Hospital for Children at IU Healthמיקוד מאומתאינדיאנפוליס, ארצות הברית
- בית החולים ג'ונס הופקינסמיקוד מאומתבולטימור, ארצות הברית
- Boston Children's Hospitalמיקוד מאומתבוסטון, ארצות הברית
- Children's Mercy Hospital and Clinicsמיקוד מאומתקנזס סיטי, ארצות הברית
- Northwell Health Systemמיקוד מאומתNew Hyde Park, United States
- Hassenfeld Children's Hospital at NYU Langoneמיקוד מאומתניו יורק, ארצות הברית
- New York-Presbyterian / Columbia University Irving Medical Centerמיקוד מאומתניו יורק, ארצות הברית
- The Childrens Hospital at Montefiore Albert Einstein School of Medicineמיקוד מאומתהברונקס, ארצות הברית
- Atrium Health Carolinas Medicalמיקוד מאומתדרהאם, ארצות הברית
שאלות נפוצות
What is Alagille syndrome?
It's a rare genetic condition that can affect the liver, heart, and other body parts, causing issues like yellow skin, poor growth, and severe itching.
What is odevixibat?
Odevixibat is a medicine being studied to help people with Alagille syndrome, especially to reduce severe itching. It's already approved in the US for this purpose in some children.
כמה זמן יימשך המחקר?
The main treatment period is about 72 weeks for some participants and 12 weeks for babies. There might be an option to continue longer if the medicine is helping.
האם אצטרך לשלם כדי להשתתף במחקר?
No, you should not have to pay for the study medicine or related tests. Any travel costs might be covered; ask the study team for details.
Can I stop being in the study if I change my mind?
Yes, you can choose to leave the study at any time without having to give a reason, and it won't affect your future medical care.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
דיון
דיון קהילתי
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