EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
The EXACT study is a research trial exploring a new medication called Exaluren for Alport Syndrome, a genetic condition that affects the kidneys. This study aims to find out if Exaluren can help protect the kidneys and is safe to use. It's a 'blinded' study, meaning neither you nor your doctors will know if you're receiving the new medicine or a dummy treatment (placebo) at first. This helps us get clear results. The study involves a small group of people aged 12 and over who have specific genetic changes linked to their Alport Syndrome. Participants will take the medicine daily by injection under the skin for about 8 months, with follow-up appointments afterwards.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
The EXACT study is a clinical trial that aims to test a new medicine called Exaluren for people living with Alport Syndrome. Alport Syndrome is a inherited condition that primarily affects the kidneys, but can also impact hearing and eyesight. It happens because of changes in certain genes that are important for healthy kidney filters.
Researchers want to see if Exaluren can help slow down the kidney damage caused by Alport Syndrome. For this study, they are specifically looking for people whose Alport Syndrome is caused by particular types of genetic changes, called 'nonsense mutations,' in genes named COL4A3, COL4A4, or COL4A5. By carefully studying how Exaluren works in these patients, scientists hope to find a new treatment option.
This study is important because new treatments are always needed to help manage conditions like Alport Syndrome and improve the lives of those affected. The information gathered from this trial will help us understand if Exaluren is an effective and safe option for people with this specific type of Alport Syndrome.
النقاط الرئيسية
- The study tests a new medicine, Exaluren, for Alport Syndrome.
- It's for people aged 12 and older with specific genetic changes.
- Participation involves daily injections and regular hospital visits.
- The study lasts about 10 months, including screening and follow-up.
- You will initially receive either the drug or a placebo, then switch to the drug.
- سيتم مراقبة صحتك عن كثب طوال الوقت.
من قد يكون مؤهلاً؟
To join this study, you would need to have a confirmed diagnosis of Alport Syndrome (specifically X-linked or autosomal recessive types) and have certain genetic changes (called nonsense mutations) in your COL4A3, COL4A4, or COL4A5 genes. You also need to be aged 12 or over. Your kidneys should be working reasonably well, but you must have a certain level of protein in your urine, which indicates kidney damage, and you should already be taking standard medication for your kidneys (like ACE inhibitors or ARBs) for at least 12 weeks before starting the study.
There are also some reasons why you might not be able to join. For example, if you have had an organ transplant, have severe liver disease, or have ever needed dialysis. If your kidneys have suddenly gotten worse recently, or if you experience active dizziness, you also wouldn't be able to take part.
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 I have a confirmed diagnosis of Alport Syndrome (X-linked or autosomal recessive)?
- Have I had a genetic test showing a 'nonsense mutation' in COL4A3, COL4A4, or COL4A5?
- Am I 12 years old or older?
- Am I currently taking an ACE inhibitor or ARB for my kidneys, and have I been on it for at least 3 months?
- Have I ever had an organ transplant or severe liver disease?
- Have I had acute kidney injury or needed dialysis in the last month?
ماذا يتضمن المشاركة؟
If you decide to take part, you'll first have a 'screening period' lasting up to 6 weeks to check if you're suitable. Once confirmed, you'll start the 'treatment period' which lasts about 32 weeks (around 8 months). For the first half (16 weeks), you'll be randomly given either Exaluren or a dummy treatment (placebo). Neither you nor your doctor will know which one you are getting. For the second half (another 16 weeks), everyone will receive Exaluren.
The medication is given daily as an injection under the skin. After your treatment period, there will be a 'follow-up period' of 4 weeks to see how you're doing. Throughout the study, you'll have regular hospital visits for check-ups, blood tests, and other assessments to monitor your health and how the treatment is working. The total time you'll be involved in the study, from screening to the end of follow-up, will be around 10 months.
المخاطر والفوائد المحتملة
المواقع (11)
- جامعة كاليفورنيا، لوس أنجلوسالرمز البريدي المؤكدلوس أنجلوس، الولايات المتحدة· لم يبدأ التوظيف بعد
- جامعة ستانفوردالرمز البريدي المؤكدستانفورد، الولايات المتحدة· لم يبدأ التوظيف بعد
- Denver Nephrologists PC, Colorado Kidney Care PCالرمز البريدي المؤكددنفر، الولايات المتحدة· التوظيف
- جامعة مينيسوتاالرمز البريدي المؤكدمينيابوليس، الولايات المتحدة· لم يبدأ التوظيف بعد
- مركز هاكنساك الطبي الجامعيالرمز البريدي المؤكدهاكنساك، الولايات المتحدة· لم يبدأ التوظيف بعد
- جامعة بنسلفانياالرمز البريدي المؤكدفيلادلفيا، الولايات المتحدة· لم يبدأ التوظيف بعد
- Renal Associates, P.A.الرمز البريدي المؤكدسان أنطونيو، الولايات المتحدة· لم يبدأ التوظيف بعد
- Alder Hey Children's NHS Foundation Trustالرمز البريدي المؤكدليفربول، المملكة المتحدة· لم يبدأ التوظيف بعد
- Royal Free Hospitalالرمز البريدي المؤكدلندن، المملكة المتحدة· التوظيف
- مستشفى جريت أورموند ستريتالرمز البريدي المؤكدلندن، المملكة المتحدة· التوظيف
- مستشفى رويال مانشستر للأطفالالرمز البريدي المؤكدمانشستر، المملكة المتحدة· لم يبدأ التوظيف بعد
أسئلة شائعة
What is Alport Syndrome?
Alport Syndrome is a genetic condition that mostly affects the kidneys, but can also impact hearing and eyesight. It's caused by faulty genes that help make an important part of your kidney filters.
What does 'blinded study' mean?
It means for the first part of the study, neither you nor your doctor will know if you're taking the new medicine (Exaluren) or a dummy treatment (placebo). This helps make the study results more reliable.
كيف يتم إعطاء الدواء؟
The medicine, Exaluren, is given as a daily injection under the skin (like an insulin injection).
ما هي المدة التي سأكون مشاركًا فيها في الدراسة؟
From the initial checks to the final follow-up, you would be involved for about 10 months in total.
هل يمكنني التوقف عن المشاركة إذا أردت؟
Yes, you can leave the study at any time, and you don't have to give a reason. Your regular medical care will not be affected.
كيفية معرفة المزيد
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
هل أنت مهتم بالمشاركة؟
نقاش
مناقشة المجتمع
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