Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
This research study is looking at a new treatment called ARO-ATXN2 for adults diagnosed with Spinocerebellar Ataxia Type 2 (SCA2). SCA2 is a genetic condition that affects balance and coordination. The main goal of this 'Phase 1' study is to check if the ARO-ATXN2 injection is safe for people, how well their bodies tolerate it, and how the medicine works within the body. Participants will be randomly chosen to receive either a single dose of ARO-ATXN2 or a placebo (a dummy injection that looks the same but contains no active medicine). Researchers will then carefully monitor them to understand any effects of the treatment. This is an early stage of testing new medications.
במבט חטוף
על מה המחקר הזה?
This study is an early investigation into a new potential treatment for a condition called Spinocerebellar Ataxia Type 2, or SCA2. SCA2 is a genetic condition that affects the brain, causing problems with movement, balance, and coordination. It happens because of a specific genetic change, and this study is inviting people who have this particular genetic change and are experiencing symptoms.
The new treatment being tested is an injection called ARO-ATXN2. The main purpose of this study, often called a 'Phase 1' study, is to find out if the ARO-ATXN2 injection is safe for people with SCA2 and how well their bodies can cope with it. Researchers also want to understand how the drug moves through the body and what effects it might have at a cellular level.
Participants in this study will be randomly assigned to receive either the ARO-ATXN2 injection or a placebo injection. A placebo is a treatment that looks exactly like the real thing but contains no active medication. This helps researchers compare the effects of the active drug to no treatment at all. All participants will be closely monitored after receiving their injection.
נקודות עיקריות
- It's an early-stage (Phase 1) study for Spinocerebellar Ataxia Type 2 (SCA2).
- It's testing a new injection called ARO-ATXN2 for safety and how the body handles it.
- Participants will receive either the active drug or a placebo (dummy injection).
- The treatment is given via a single injection into the spinal fluid.
- Close medical monitoring will happen throughout the study.
מי עשוי להיות זכאי?
To join this study, you must be an adult aged between 18 and 65 years old. You need to have a confirmed diagnosis of Spinocerebellar Ataxia Type 2 (SCA2) and carry a specific genetic marker (at least 33 CAG repeats in the ATXN2 gene), which will be checked from your medical records or a genetic test.
There are also some health conditions that would prevent you from taking part. For instance, if you are pregnant or breastfeeding, you cannot join. People with certain uncontrolled health issues like very high blood pressure, serious heart, liver, or kidney disease, or specific infections like HIV or hepatitis B or C, would not be suitable.
Additionally, if you’ve had stem cell therapy previously, have a learning disability, or any problem that would make a procedure called a 'lumbar puncture' unsafe, you wouldn't be able to participate. If you're a woman who could become pregnant, you would need to agree to use effective birth control during and for some time after the study.
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 an adult aged 18 to 65?
- Do you have a confirmed diagnosis of SCA2?
- Are you able to use effective birth control if you are a woman of childbearing potential?
- Do you have any serious health conditions like uncontrolled high blood pressure or severe heart, liver, or kidney problems?
- Have you ever received stem cell therapy?
- Are you able to safely have a procedure called a 'lumbar puncture'?
מה כרוך בהשתתפות?
If you decide to take part in this study, you would first go through a screening process to make sure you're eligible. If you are, you would then be randomly assigned to receive either the ARO-ATXN2 injection or a placebo (a dummy injection). This will be a single injection administered through a 'lumbar puncture', which is a procedure where a small needle is inserted into your lower back to deliver the medicine into the fluid around your spinal cord.
After the injection, you would have several follow-up appointments. These visits will involve various tests and checks to monitor your health, look for any side effects, and see how your body is reacting to the treatment. This includes checking your general health, possibly having blood tests, and further assessments of your SCA2 symptoms. The total duration of your involvement in the study would be outlined by the study team.
סיכונים ותועלות פוטנציאליים
Locations (16)
- Research Site 8מיקוד מאומתSydney, Australia· גיוס
- אתר מחקר 7מיקוד מאומתמלבורן, אוסטרליה· גיוס
- Research Site 2מיקוד מאומתמונטריאול, קנדה· גיוס
- אתר מחקר 1מיקוד מאומתמונטריאול, קנדה· גיוס
- אתר מחקר 9מיקוד מאומתאדמונטון, קנדה· גיוס
- אתר מחקר 15מיקוד מאומתפריז, צרפת· גיוס
- אתר מחקר 13מיקוד מאומתטיבינגן, גרמניה· גיוס
- אתר מחקר 14מיקוד מאומתאולם, גרמניה· גיוס
- אתר מחקר 16מיקוד מאומתמילאנו, איטליה· גיוס
- אתר מחקר 4מיקוד מאומתAuckland, New Zealand· גיוס
- אתר מחקר 3מיקוד מאומתChristchurch, New Zealand· גיוס
- אתר מחקר 10מיקוד מאומתברצלונה, ספרד· גיוס
שאלות נפוצות
What is Spinocerebellar Ataxia Type 2 (SCA2)?
SCA2 is a genetic condition that affects the brain, causing problems with balance, coordination, and movement.
מהו 'פלצבו'?
A placebo is adummy treatment that looks just like the real medicine but doesn't contain any active drug. It helps researchers compare the new treatment's effects.
What is a 'lumbar puncture'?
This is a medical procedure where a thin needle is gently inserted into your lower back to deliver medicine into the fluid surrounding your spinal cord.
האם אדע אם אני מקבל את התרופה האמיתית או את הפלצבו?
No, you won't know at the time. This is done so that the results are not influenced by what you expect. The researchers also won't know immediately.
כמה זמן אהיה במחקר?
The study team will tell you the exact length of your participation, including follow-up visits, during the consent process.
כיצד לגלות עוד
צג רפואי
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
מעוניינים להשתתף?
דיון
דיון קהילתי
מופעל על ידי הפורום שלנו ב community.patient.info. אנא היו מכבדים — זה אינו ייעוץ רפואי.