A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study is looking for young people aged 12-17 who have a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). The main goal is to find out if a new medicine, called pegcetacoplan, is safe and works well for teenagers with PNH. Researchers also want to understand how the body uses and processes this medicine. Participants will receive the study medicine, pegcetacoplan, as an injection under the skin at home, twice a week. The study involves a screening period, followed by a treatment period of 16 weeks. Some participants might then join a longer-term study, while others will have a follow-up period. This is an important step in helping to develop better treatments for PNH.
במבט חטוף
על מה המחקר הזה?
You've been diagnosed with Paroxysmal Nocturnal Hemoglobinuria, or PNH. This is a very rare blood condition where your red blood cells are destroyed too quickly, which can cause anaemia and other problems. Doctors and scientists are always looking for better ways to treat PNH, especially for younger people.
This study is testing a new medicine called pegcetacoplan. It's designed to see if it can help improve the symptoms of PNH and how your body reacts to it. The researchers want to make sure it's safe and that it works well for teenagers aged 12 to 17, as their bodies can respond differently to medicines compared to adults.
Taking part in a study like this helps doctors learn more about PNH and potential new treatments. Your involvement could help others with PNH in the future. It’s important to remember that this medicine is still being studied, and isn't yet widely available.
נקודות עיקריות
- This study is for teenagers (12-17) with PNH.
- It's testing a new medicine called pegcetacoplan.
- The medicine is given as an injection under the skin twice a week at home.
- The main treatment part lasts 16 weeks, after screening.
- You'll be closely monitored for safety and how the medicine works.
- Your involvement could help others with PNH in the future.
מי עשוי להיות זכאי?
To be considered for this study, you need to be between 12 and 17 years old and weigh at least 20 kilograms (about 3 stone 2 pounds). You must have a confirmed diagnosis of PNH.
Even if you're already taking other PNH medicines (like eculizumab or ravulizumab), you might still be able to join, especially if you still have anaemia symptoms. If you're not on those medicines, you'll need to have signs of anaemia, which means low red blood cell counts and certain blood test results.
There are also some reasons you wouldn't be able to join. For example, if you're pregnant or breastfeeding, or if you have a history of certain medical conditions like hereditary complement deficiency (a different immune system problem), a bone marrow transplant, or meningococcal disease (like meningitis). You also can't have a known problem with how your body handles fructose (a type of sugar).
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 between 12 and 17 years old?
- Do you have a confirmed diagnosis of PNH?
- Do you weigh at least 20 kilograms (about 3 stone 2 pounds)?
- האם את לא בהריון או מניקה?
- Do you NOT have a past history of meningitis or certain other conditions?
מה כרוך בהשתתפות?
If you decide to take part, the first step is a 4-week screening period where doctors will do tests to see if the study is right for you. If you're currently on a different PNH medicine, there might be an extra 4-week period to carefully switch you over.
After screening, you'll enter a 16-week treatment period. During this time, you'll receive the study medicine, pegcetacoplan, twice a week. It's given as an injection under the skin, which you or your caregiver will be taught to do at home. This involves two small needles placed into the fatty layer just under your skin, through which the medicine flows.
Once the 16-week treatment is over, you might have the option to join a longer-term study to keep receiving the treatment, or you'll have a 2-month follow-up period where doctors will monitor your health.
סיכונים ותועלות פוטנציאליים
מיקומים (12)
- Children's Hospital of Atlantaמיקוד מאומתאטלנטה, ארצות הברית· גיוס
- Motol University Hospitalמיקוד מאומתפראג, צ'כיה· הושלם
- Robert-Debré Hospital Parisמיקוד מאומתפריז, צרפת· עדיין לא מגייסים
- Hospital Ampangמיקוד מאומתAmpang, Malaysia· גיוס
- Radboud University Hospital Nijmegenמיקוד מאומתNijmegen, Netherlands· הושלם
- University Medical Center Utrechtמיקוד מאומתאוטרכט, הולנד· גיוס
- University Children's Hospitalמיקוד מאומתBelgrade, Serbia· גיוס
- University Hospital Vall d'Hebronמיקוד מאומתברצלונה, ספרד· גיוס
- University Hospital 12 de Octubreמיקוד מאומתמדריד, ספרד· גיוס
- Phramongkutklao Hospital and College of Medicineמיקוד מאומתBangkok, Thailand· הושלם
- Maharaj Nakorn Chiang Mai hospitalמיקוד מאומתChiang Mai, Thailand· גיוס
- St. Mary's Hospitalמיקוד מאומתלונדון, הממלכה המאוחדת· הושלם
שאלות נפוצות
What is PNH?
PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare blood disorder where your immune system mistakenly attacks and destroys your red blood cells.
What is pegcetacoplan?
Pegcetacoplan is the medicine being tested in this study. It's designed to help calm down the part of the immune system that causes damage in PNH.
How will I get the medicine?
The medicine is given as an injection under your skin, called a subcutaneous infusion, twice a week at home. You or a caregiver will be taught how to do this safely.
כמה זמן יימשך המחקר?
The main treatment part of the study lasts for 16 weeks, after a screening period of about 4 weeks. There might be options for longer treatment or follow-up after that.
האם אני יכול להפסיק להשתתף אם אני רוצה?
Yes, absolutely. You can leave the study at any time, for any reason, and your regular medical care won't be affected.
כיצד לגלות עוד
Apellis Clinical Trial Information Line
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