HALOS: دراسة السلامة والتحمل والحركية الدوائية والديناميكية الدوائية لجرعات متصاعدة متعددة من ION582 لدى المشاركين المصابين بمتلازمة أنجلمان
The HALOS study is investigating a new medication called ION582 for people with Angelman syndrome. The main goals are to check if the medicine is safe at different doses and how well people tolerate it. Researchers also want to understand how the medicine works in the body and its effects on Angelman syndrome. This study is split into several parts and will involve around 70 participants, ranging from babies to adults up to 50 years old. A recent change means that younger children (under 2 years old) with Angelman syndrome can now join at some locations. If you're interested, it's best to speak with your doctor or the study team for more details.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
The HALOS study is a clinical trial focused on a new experimental medicine called ION582, designed for individuals with Angelman syndrome. Angelman syndrome is a complex genetic condition that affects development, often causing learning difficulties, problems with speech, movement, and sometimes seizures. There is currently no cure, so new treatments are really important.
This study has several aims. First, it wants to find out if ION582 is safe to use at different dose levels. This is always the first step for any new medicine. Secondly, researchers will be looking at how the body processes the medicine – how it's absorbed, distributed, and eventually leaves the body. Finally, they want to understand if ION582 has any positive effects on the symptoms of Angelman syndrome.
This is a 'Phase 1-2a' study, meaning it's an early-stage study focusing on safety, but also looking for the first signs that the treatment might work. It's an 'open-label' study, which means both the participants and the medical team will know who is receiving the study medicine. The study will happen in different stages, with some participants potentially being in the study for several years to see the long-term effects of the medicine.
النقاط الرئيسية
- This study is testing a new medicine, ION582, for Angelman syndrome.
- It aims to check the safety and how well people tolerate different doses.
- Participants can range from babies to adults up to 50 years old.
- The medicine is given through injections into the spinal fluid.
- Participation could involve regular hospital visits over several years.
- A recent update allows some sites to recruit children under 2 years old.
من قد يكون مؤهلاً؟
To be considered for this study, individuals must have a confirmed diagnosis of Angelman syndrome due to a specific gene change (called a 'UBE3A deletion' or 'UBE3A mutation'). The study is open to both males and females between birth and 50 years of age. For those under 18, a parent or legal guardian would need to agree to their participation.
It's important that any other treatments for Angelman syndrome, such as seizure medications, sleep aids, or special diets, have been stable for at least three months before joining. This helps the researchers understand the effects of the new study medicine more clearly. One unique request is that participants (or their guardians) agree not to share personal or study information on social media until the study is officially finished.
However, some people would not be able to take part. This includes those whose Angelman syndrome is caused by a different genetic change (called 'paternal uniparental disomy' or 'imprinting defect'). Also, if someone has other serious medical conditions that the doctor thinks would make taking part unsafe, or if they have poorly controlled seizures, they would not be eligible. People who have certain bone or bleeding problems, or have had gene therapy in the past, also cannot join. If you've been vaccinated against COVID-19, that's absolutely fine, you can still participate.
هل يمكن أن تناسبك هذه الدراسة؟
أجب عن هذه الأسئلة السريعة لترى ما إذا كنت مؤهلاً. هذا دليل فقط - الفريق البحثي هو الذي يتخذ القرار النهائي.
- Do you (or the person you care for) have a confirmed Angelman syndrome diagnosis due to a UBE3A deletion or mutation?
- Are you (or the person you care for) between birth and 50 years old?
- Have any other Angelman syndrome treatments been stable for at least 3 months?
- Are there any other serious health conditions that might make participating unsafe?
- Have you (or the person you care for) had gene therapy in the past?
- Are you able to commit to not sharing study information on social media until complete?
ماذا يتضمن المشاركة؟
If you join the HALOS study, you would first go through a screening period to check if you meet all the requirements. The study medicine, ION582, is given directly into the fluid around the spinal cord (this is called an 'intrathecal' injection). This is similar to how some pain medications are given.
The study is divided into several parts. Part 1 involves receiving increasing doses of the medicine over 13 weeks, followed by at least 12 weeks of monitoring. If you complete Part 1, you might then move to Part 2, which involves receiving the medicine for 49 weeks, again followed by a monitoring period. For those who complete Part 2, there’s an option to continue receiving the medicine for up to another three years, followed by a final monitoring period. This means participating could involve regular hospital visits over several years, with check-ups, assessments, and the medicine being given. The full duration of the study for an individual could extend for several years.
المخاطر والفوائد المحتملة
المواقع (11)
- مستشفى رادي للأطفالالرمز البريدي المؤكدسان دييغو، الولايات المتحدة
- Colorado Children's Hospital Research Instituteالرمز البريدي المؤكدأورورا، الولايات المتحدة
- مركز جامعة روش الطبيالرمز البريدي المؤكدشيكاغو، الولايات المتحدة
- مستشفى بوسطن للأطفالالرمز البريدي المؤكدبوسطن، الولايات المتحدة
- University of North Carolina at Chapel Hill School of Medicineالرمز البريدي المؤكدCarrboro, United States
- Texas Children's Hospitalالرمز البريدي المؤكدهيوستن، الولايات المتحدة
- Sydney Children's Hospital, Kids Cancer Centreالرمز البريدي المؤكدراندويك، أستراليا
- Necker-Enfants Malades Hospitalالرمز البريدي المؤكدباريس، فرنسا
- مركز شيبا الطبيالرمز البريدي المؤكدرمات غان، إسرائيل
- Azienda Ospedaliera Universitaria Pisanaالرمز البريدي المؤكدPisa, Italy
- STRONG Group University of Oxfordالرمز البريدي المؤكدأوكسفورد، المملكة المتحدة
أسئلة شائعة
What is Angelman syndrome?
Angelman syndrome is a genetic condition affecting development, causing learning difficulties, speech and movement problems, and sometimes seizures.
What is ION582?
ION582 is an experimental medicine being tested in this study to see if it can help people with Angelman syndrome.
كيف يتم إعطاء الدواء؟
The medicine is given directly into the fluid around the spinal cord through an injection, similar to some pain procedures.
Can young children join the study?
Yes, a recent change means that children under 2 years old with Angelman syndrome can now join at some study locations.
What is the study called?
The study is called HALOS.
كيفية معرفة المزيد
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
نقاش
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