An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH)
This research study is testing a new medicine called IKT-001 for people in the UK who have a type of high blood pressure in their lungs, known as Pulmonary Arterial Hypertension (PAH). This condition makes it hard for blood to flow through the lungs, causing breathlessness and tiredness. The study aims to find out if IKT-001 is effective and safe. Participants will either receive the new medicine or a placebo (a dummy treatment with no active drug) to compare the effects. The study is organised carefully to make sure results are reliable, involving many hospitals and keeping both patients and doctors unaware of who gets which treatment until the end.
במבט חטוף
על מה המחקר הזה?
This study is designed to learn more about a new medicine called IKT-001 for a condition known as Pulmonary Arterial Hypertension (PAH). PAH is a serious condition where the blood vessels in the lungs become narrowed, making it harder for blood to flow through. This causes high blood pressure in the arteries leading to the lungs, which can make people feel very breathless and tired. The aim of this research is to see if IKT-001 can help improve the symptoms and overall health of adults living with this condition.
This is a 'Phase 3' study, which means the new medicine has already been tested in smaller groups of people and shown some promise. Now, researchers want to test it on a larger number of patients to confirm if it truly works and is safe. Some people in the study will receive IKT-001, while others will get a placebo – a dummy treatment that looks just like the real medicine but contains no active drug. This helps scientists fairly compare the effects of the new medicine.
To ensure the results are trustworthy, it's a 'double-blind' study. This means neither you nor your doctor will know if you're receiving IKT-001 or the placebo. It's also a 'multicenter' study, meaning it's happening in many hospitals, allowing more people to take part and providing a broader picture of the medicine's effects.
נקודות עיקריות
- This study is for adults with a specific type of lung condition called Pulmonary Arterial Hypertension (PAH).
- It's testing a new medicine, IKT-001, against a dummy treatment (placebo).
- Participation involves regular clinic visits for health checks and taking study medication.
- Neither you nor your doctor will know if you're getting the active drug or the placebo.
- You might benefit from the new treatment or help advance medical knowledge.
- אתה יכול להפסיק להשתתף בכל עת מבלי להשפיע על הטיפול הרפואי שלך.
מי עשוי להיות זכאי?
To be part of this study, you need to be an adult between 18 and 75 years old and have a specific diagnosis of Pulmonary Arterial Hypertension (PAH), known as WHO Group 1. This includes types like PAH that happens without a clear cause, PAH that runs in families, or PAH linked to certain drugs, connective tissue diseases, or a repaired heart condition present since birth.
You also need to be on stable treatment for your PAH, such as specific medications like endothelin receptor antagonists, phosphodiesterase-5 inhibitors, prostacyclins, or soluble guanylate cyclase stimulators, for at least 3 months before joining. Your body mass index (BMI) should be between 18.5 and 35. You'll need to have had specific tests to measure the pressure in your lungs that show your condition fits the study requirements, and you should be able to walk a certain distance in six minutes (between 100 and 475 metres).
There are also reasons why you might not be able to join. For example, if you have other types of PAH (WHO Groups 2, 3, 4, or 5) or specific Group 1 types like HIV-associated PAH. You would also be excluded if you have very high or very low blood pressure, have fainted recently, or have certain heart problems or a history of specific heart rhythm issues. Having some implanted heart monitoring devices would also prevent you from taking 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.
- האם אתה בין גיל 18 ל-75?
- Do you have a diagnosis of WHO Group 1 Pulmonary Arterial Hypertension (PAH)?
- Have you been on stable PAH medication for at least 3 months?
- Do you have any significant heart problems (apart from PAH) or very high/low blood pressure?
- Can you attend regular clinic appointments for the duration of the study?
מה כרוך בהשתתפות?
If you decide to take part in this study, you will be in it for a while, and it will involve several visits to the study clinic. First, there's a 'screening period' to make sure you're suitable for the study based on your health and tests. After this, if you qualify, you will start the main treatment phase.
During the study, you'll be given either the new medicine, IKT-001, or a placebo (a dummy drug). You'll need to take this medicine regularly as instructed. You'll have scheduled visits to the clinic for various health checks. These will likely include physical examinations, blood tests, heart checks like ECGs, and repeat lung pressure measurements. You may also be asked to do walking tests (like the 6-minute walk test) to see how your symptoms are changing.
The study team will carefully monitor your health and any side effects you might experience. It's important to attend all your appointments and report any new symptoms or concerns. The total length of your participation, including follow-up, will be explained fully by the study team, but these types of studies can often last for many months.
סיכונים ותועלות פוטנציאליים
מיקומים (43)
- Arizona Pulmonary Specialists, LTDמיקוד מאומתסקוטסדייל, ארצות הברית· גיוס
- Cedars Sinaiמיקוד מאומתבוורלי הילס, ארצות הברית· גיוס
- Jeffrey S. Sager, MD Medical Corporationמיקוד מאומתSanta Barbara, United States· גיוס
- Lundquist at Harbor UCLAמיקוד מאומתטורנס, ארצות הברית· גיוס
- אוניברסיטת קולורדו קמפוס הרפואי אנסוץמיקוד מאומתאורורה, ארצות הברית· גיוס
- המרכז הרפואי של אוניברסיטת קנזסמיקוד מאומתקנזס סיטי, ארצות הברית· גיוס
- Norton Healthcareמיקוד מאומתלואיוויל, ארצות הברית· גיוס
- מרכז רפואי טאפטסמיקוד מאומתבוסטון, ארצות הברית· גיוס
- בית הספר לרפואה של אוניברסיטת וושינגטוןמיקוד מאומתסנט לואיס, ארצות הברית· גיוס
- NYU Langone Healthמיקוד מאומתניו יורק, ארצות הברית· גיוס
- Westchester Medical Centerמיקוד מאומתValhalla, United States· גיוס
- אוניברסיטת דיוקמיקוד מאומתדרהאם, ארצות הברית· גיוס
שאלות נפוצות
מהי יתר לחץ דם ריאתי עורקי (PAH)?
PAH is a rare condition where the blood vessels in your lungs become narrowed, making it hard for blood to flow through. This raises the pressure in your lungs and can cause breathlessness and tiredness.
What is IKT-001?
IKT-001 is a new medicine being tested in this study to see if it can help people with a specific type of high blood pressure in their lungs (PAH).
מהו 'פלצבו'?
A placebo is a dummy treatment that looks exactly like the study medicine but contains no active drug. It helps compare the effects of the real medicine fairly.
Will I know if I am getting the real medicine or the placebo?
No, this is a 'double-blind' study, meaning neither you nor your doctor will know whether you are receiving IKT-001 or the placebo until the study is over.
כמה זמן אהיה במחקר?
The full length of the study, including all visits and follow-up, will be explained by the study team if you are eligible and interested in participating.
כיצד לגלות עוד
Medical Director, Inhibikase Therapeutics
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.