A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
This research study is investigating an experimental combination treatment involving two investigational drugs: pozelimab and cemdisiran. It's for adults who have a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). The main goal is to understand how safe this combination treatment is and how well it works over a long period. Researchers will also look for any side effects, how the drugs move through the body, and if the body makes antibodies that might affect the treatment. This is a Phase 3 study, meaning these drugs have already been tested in earlier stages, and now more information is needed on their long-term use.
במבט חטוף
על מה המחקר הזה?
This study is about a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. PNH can cause problems like tiredness, blood clots, and damage to red blood cells. Researchers are looking into a new way to treat PNH using two new drugs together: pozelimab and cemdisiran. These drugs are not yet available as regular treatments, so they are being tested in this study.
The main purpose of this study is to find out more about how safe and how effective this pozelimab and cemdisiran combination is for people with PNH when taken over a longer period. The study aims to gather important information that could help these drugs become available to more people in the future.
The study will look at several key questions. For example, it will check for any side effects, how well the treatment helps with PNH symptoms, and how the drugs behave in the body. They will also investigate if people's bodies develop a response to the drugs, which could make them less effective.
נקודות עיקריות
- This study evaluates two new drugs for PNH: pozelimab and cemdisiran.
- It aims to understand the long-term safety and effectiveness of this combination.
- Only adults (18+) with PNH can take part.
- Two main groups are eligible: those from a previous study and PNH patients with a specific gene change (C5 polymorphism).
- Participation involves clinic visits, taking study medication, and health monitoring.
- אתה יכול להפסיק להשתתף בכל עת.
מי עשוי להיות זכאי?
This study is looking for adult patients (aged 18 and over) with PNH. Generally, there are two main groups of people who might be able to join. One group includes patients who have already been taking part in a similar earlier study (called the 'parent study') and completed it without stopping the treatment.
The second group includes PNH patients who have a specific gene change (called a 'C5 polymorphism') that means their current PNH treatments (like eculizumab or ravulizumab) might not be working as well as expected. For these patients, doctors would need to confirm their PNH diagnosis with a special blood test and show that their condition is currently active, with certain blood markers being high.
There are also some reasons why someone might not be able to join. For example, if they have had an organ or bone marrow transplant, or if the research doctors believe that joining the study might not be safe for them or might interfere with the study's goals. Also, all participants must be up-to-date with specific vaccinations, such as for meningococcal disease.
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 בן 18 או יותר?
- Do you have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH)?
- Have you been fully vaccinated, including for meningococcal disease?
- If you have a C5 polymorphism, is your PNH currently active with specific high blood markers?
- Are you able and willing to attend regular clinic visits and follow study instructions?
מה כרוך בהשתתפות?
If you join this study, you will receive the experimental combination of pozelimab and cemdisiran. The study involves regular visits to the clinic, where the research team will monitor your health, check for any side effects, and take blood samples to see how the drugs are working and how much of them are in your blood. You will also need to comply with specific vaccination requirements, particularly for meningococcal disease. The exact number of visits, assessments, and the total duration will be carefully explained to you by the study team if you are considered for participation.
סיכונים ותועלות פוטנציאליים
מיקומים (48)
- A Beneficencia Portuguesa de Sao Paulo, BP Miranteמיקוד מאומתSão Paulo, Brazil· גיוס
- בית החולים הכללי של טורונטומיקוד מאומתטורונטו, קנדה· גיוס
- Hospital Pablo Tobon Uribeמיקוד מאומתMedellín, Colombia· גיוס
- Semmelweis University/Semmelweis Egyetemמיקוד מאומתבודפשט, הונגריה· גיוס
- Amrita Institute of Medical Sciences (AIMS) and Research Centre Aimsמיקוד מאומתKochi, India· גיוס
- K J Somaiya Super Specialty Hospital & Research Centreמיקוד מאומתMumbai, India· גיוס
- Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campusמיקוד מאומתNew Delhi, India· גיוס
- Postgraduate Institute of Medical Education & Researchמיקוד מאומתChandigarh, India· גיוס
- Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)מיקוד מאומתJaipur, India· גיוס
- Aou Careggiמיקוד מאומתפירנצה, איטליה· גיוס
- SC Hematology, AOU Città della Salute e della Scienza di Torinoמיקוד מאומתTorino, Italy· גיוס
- Japanese Red Cross Aichi Medical Center Nagoya Daini Hospitalמיקוד מאומתNagoya, Japan· נמשך
שאלות נפוצות
What is PNH?
PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare, long-lasting blood condition where red blood cells are destroyed too early, which can lead to tiredness, blood clots, and other problems.
Are pozelimab and cemdisiran available as regular medicines?
No, these are experimental drugs, meaning they are still being tested in studies like this one and are not yet approved for general use.
מה המשמעות של 'שלב 3' עבור מחקר?
Phase 3 means the drugs have already been tested in smaller groups, and now researchers need to study them in a larger group of people to confirm how safe and effective they are over a longer time.
Will I get 'placebo' or dummy treatment?
The information provided suggests all participants in this extension study will receive the active study drugs, pozelimab and cemdisiran. There is no mention of a placebo.
מי משלם עבור המחקר הזה?
This study is sponsored by a pharmaceutical company, Regeneron Pharmaceuticals, Inc., who are developing these drugs.
כיצד לגלות עוד
Clinical Trials Administrator
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