מחקר הארכה לטווח ארוך להערכת הבטיחות והיעילות לטווח ארוך של טיפול בפגטיבטינאז במשתתפים בגילאי 5 עד 65 שנים עם הומוציסטינוריה קלאסית (HCU) (ENSEMBLE)
This study, called ENSEMBLE, is investigating a medicine named pegtibatinase for a condition called Homocystinuria (HCU). It's a long-term study checking the safety and how well pegtibatinase works in people aged 5 to 65. Participants will be those who are already taking part in a previous pegtibatinase study (COMPOSE) or have finished 24 weeks of treatment in another study (HARMONY). The main goal is to keep track of patients over an extended period to understand the full effects of the treatment. This helps researchers gather important information about this new medicine as a potential long-term option for HCU.
במבט חטוף
על מה המחקר הזה?
This study, called ENSEMBLE, is looking into a medication named pegtibatinase. It’s designed for people who have a rare genetic condition called Homocystinuria, often shortened to HCU. This condition affects how the body breaks down certain proteins, leading to a build-up of harmful substances. Researchers are interested in seeing how safe and effective pegtibatinase is as a long-term treatment for those with HCU.
Since this is an 'extension' study, it means that the people who can join have already been part of an earlier study looking at the same medication. By continuing to follow these participants for a longer time, the researchers can gather much more information about the treatment's effects. This includes understanding if the benefits continue over many years and if there are any long-term side effects.
The information gathered from this study is very important because it helps scientists and doctors understand if pegtibatinase could be a valuable and safe treatment option for people with HCU for a considerable period. This is a crucial step in developing new medicines that can genuinely improve the lives of patients.
נקודות עיקריות
- Study for Homocystinuria (HCU) patients aged 5-65.
- Investigates the long-term safety and effectiveness of pegtibatinase.
- Only for those already in specific previous pegtibatinase studies.
- Involves regular medication (self-administered) and health checks over 2 years.
- Helps understand if pegtibatinase is a good long-term HCU treatment.
- ההשתתפות היא בהתנדבות, ואתה יכול לפרוש בכל עת.
מי עשוי להיות זכאי?
To be able to join this study, you must be between 5 and 65 years old. The study is specifically for people who are already taking part in another study called COMPOSE investigating pegtibatinase. Alternatively, you can join if you have completed 24 weeks of treatment in the HARMONY study, which also looked at pegtibatinase or a placebo.
Unfortunately, you cannot join this particular study if you had to stop an earlier pegtibatinase study because of a serious side effect or if your body couldn't tolerate the treatment. This is to ensure the safety of participants already known to have had difficulties with the medication.
Both males and females are welcome to participate, as long as they meet the other criteria. The researchers want to get a broad understanding of how the treatment works across different people.
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.
- Are you between 5 and 65 years old?
- Are you currently in the COMPOSE study or have you finished the 24-week treatment part of the HARMONY study?
- Have you NOT had to stop a previous pegtibatinase study due to serious side effects or intolerance?
- Are you willing and able to potentially self-administer medication?
מה כרוך בהשתתפות?
If you join this study, you could be involved for at least 108 weeks, which is about two years. This period includes up to 104 weeks where you'll be taking the pegtibatinase medication. After you stop taking the medicine, there will be a 4-week follow-up period to check on your health and safety.
Most participants will give themselves the pegtibatinase injections, unless there's a good reason why they can't. Throughout the study, doctors will take blood samples to see how the medication is moving through your body. There's also an extra, optional part of the study (called a 'sub-study') that some people might be able to join, which looks at how much protein you can eat while on the treatment. This is an 'open-label' study, meaning everyone involved, including you and your doctors, will know you are receiving pegtibatinase.
סיכונים ותועלות פוטנציאליים
מיקומים (52)
- בית החולים לילדים פיניקסמיקוד מאומתפניקס, ארצות הברית
- Yale University School of Medicineמיקוד מאומתניו הייבן, ארצות הברית
- Emory Universityמיקוד מאומתאטלנטה, ארצות הברית
- בית החולים לילדים אן ורוברט ה. לורי בשיקגומיקוד מאומתשיקגו, ארצות הברית
- Maine Health Pediatric Specialty Careמיקוד מאומתפורטלנד, ארצות הברית
- Uncommon Curesמיקוד מאומתChevy Chase, United States
- Icahn School of Medicine at Mount Sinaiמיקוד מאומתניו יורק, ארצות הברית
- Science 37 - Virtual Siteמיקוד מאומתMorrisville, United States
- Oregon Health and Science Universityמיקוד מאומתפורטלנד, ארצות הברית
- The Children's Hospital of Philadelphiaמיקוד מאומתפילדלפיה, ארצות הברית
- University of Pittsburgh Medical Center - Children's Hospital of Pittsburghמיקוד מאומתפיטסבורג, ארצות הברית
- University of Texas Southwestern Medical Centerמיקוד מאומתדאלאס, ארצות הברית
שאלות נפוצות
What is Homocystinuria (HCU)?
Homocystinuria is a rare inherited condition that affects how your body processes certain proteins, leading to a build-up of a harmful substance called homocysteine.
What is pegtibatinase?
Pegtibatinase is the name of the new medication being studied for its potential to treat Homocystinuria.
כמה זמן אהיה במחקר?
You will be in the study for at least 108 weeks, which is just over two years. This includes taking the medication and a follow-up period.
Do I have to give myself the medicine?
Most participants will learn to give themselves the pegtibatinase injections at home, but exceptions can be made if needed.
האם אני יכול להפסיק להשתתף אם אני רוצה?
Yes, you have the right to leave the study at any time, for any reason, and it won't affect your ongoing medical care.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
מעוניינים להשתתף?
דיון
דיון קהילתי
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