REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
This research study, called REVEAL, is looking into a new medicine called ION582 for people with Angelman syndrome. Angelman syndrome is a condition present from birth that affects how a person's brain develops, often causing learning difficulties, problems with movement and balance, and seizures. The study aims to find out if ION582 is safe and effective in improving some of the symptoms of Angelman syndrome in children and adults. Participants will either receive the new medicine or a dummy medicine (placebo) for a period, followed by an opportunity for everyone to receive the active medicine for longer. Doctors will carefully monitor participants throughout the study.
במבט חטוף
על מה המחקר הזה?
This study, called REVEAL, is a really important step in understanding and potentially treating Angelman syndrome. Angelman syndrome is a challenging condition that affects many aspects of a person's life, including their learning, movement, and sleep. It's caused by a problem with a specific gene called UBE3A. This study is testing a new medicine, ION582, which is designed to help correct this gene problem.
The main goal of the study is to see if ION582 can safely make a difference for people living with Angelman syndrome. Researchers will be carefully looking at whether the medicine improves symptoms and how well people tolerate it. Because it's a 'Phase 3' study, it means the medicine has already been tested in smaller groups and now they need to gather more information from a larger number of people to confirm its effects.
Participation in this study could help advance our understanding of Angelman syndrome and potentially lead to a new treatment option for those affected. Studies like REVEAL are crucial for developing new medicines and improving the lives of individuals with rare conditions.
נקודות עיקריות
- REVEAL is a study for children and adults with Angelman syndrome.
- It's testing a new medicine called ION582.
- Participants will receive either ION582 or a dummy medicine (placebo) initially.
- The study lasts over three years, with different treatment and follow-up periods.
- It aims to see if ION582 is safe and can improve Angelman syndrome symptoms.
מי עשוי להיות זכאי?
To join this study, participants need to have Angelman syndrome caused by a specific gene fault (either a UBE3A deletion or mutation). They should be between 2 and 50 years old, depending on which group they fit into. Participants need to be generally well enough to undergo some medical procedures mentioned below.
It's important that any other medicines they are taking for Angelman syndrome, like those for seizures or sleep, have been on a steady dose for at least 8 weeks before the study starts. This helps doctors see the effect of the new medicine more clearly. Also, their caregiver or legal guardian must agree to all study procedures and not share personal medical details about the study online.
Some things might mean a person can't join. For example, if they have certain serious medical issues that might make the new medicine unsafe, or if they have brain or spine problems that would make a procedure called a 'lumbar puncture' difficult or risky. The study doctors will carefully check everyone to make sure it's safe for them 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 Angelman syndrome confirmed by a UBE3A gene test?
- Are you between 2 and 50 years old?
- Are you generally well and stable on your current medicines for Angelman syndrome?
- Is your caregiver or legal guardian able to give consent and follow study rules?
- Do you have any serious medical conditions that might make participation risky?
מה כרוך בהשתתפות?
If you join the study, it will involve several parts over a long period. First, there's a screening period of up to 28 days to check if you're suitable. Then, for about 60 weeks, you'll be in the main treatment phase where you'll either receive the study medicine (ION582) or a dummy medicine (placebo). Neither you, your family, nor the study doctors will know which one you're getting during this time.
After this, everyone who completes this first phase can enter a longer period, lasting about 25 months, where all participants will receive the active study medicine. Finally, there's a follow-up period of about 8 months after you stop taking the medicine to see how you're doing. The study involves regular visits to the clinic for assessments and to receive the medication. The total study duration, including all phases, is over three years.
סיכונים ותועלות פוטנציאליים
מיקומים (41)
- Cedars-Sinai Medical Centerמיקוד מאומתלוס אנג'לס, ארצות הברית
- UCLA Clinical & Translational Research Center (CTRC)מיקוד מאומתלוס אנג'לס, ארצות הברית
- Rady Children's Hospitalמיקוד מאומתסן דייגו, ארצות הברית
- Colorado Children's Hospital Research Instituteמיקוד מאומתאורורה, ארצות הברית
- בית החולים הלאומי לילדיםמיקוד מאומתוושינגטון די.סי., ארצות הברית
- Nicklaus Children's Hospitalמיקוד מאומתמיאמי, ארצות הברית
- Children's Healthcare of Atlantaמיקוד מאומתאטלנטה, ארצות הברית
- Rush University Medical Centerמיקוד מאומתשיקגו, ארצות הברית
- Boston Children's Hospitalמיקוד מאומתבוסטון, ארצות הברית
- Children's Mercyמיקוד מאומתקנזס סיטי, ארצות הברית
- Ichan School of Medicine at Mount Sinaiמיקוד מאומתניו יורק, ארצות הברית
- Columbia University Irving Medical Centerמיקוד מאומתניו יורק, ארצות הברית
שאלות נפוצות
What is Angelman syndrome?
Angelman syndrome is a genetic condition that causes developmental delays, problems with speech and balance, and often seizures.
What is ION582?
ION582 is an experimental medicine being tested to see if it can help improve symptoms in people with Angelman syndrome by targeting the gene involved.
מהו 'פלצבו'?
A placebo is a 'dummy' medicine that looks like the real medicine but contains no active ingredients. It helps researchers compare the effects of the new drug.
כמה זמן יימשך המחקר?
The study involves different periods, but overall it will last for a total of over three years, including treatment and follow-up.
Can I leave the study early?
Yes, you can leave the study at any time if you wish, and it won't affect your regular medical care.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
דיון
דיון קהילתי
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