A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
This research study is looking into a new medicine called crovalimab for people living with Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a very rare blood condition. The main goal is to see how safe crovalimab is and how well it works compared to a treatment already in use, called eculizumab. The study is designed for individuals who are already being treated with similar medications for their PNH. About 190 people are expected to take part. This clinical trial is an important step in understanding if crovalimab could be a new treatment option, offering potential benefits for people affected by PNH.
Em resumo
Sobre o que é este estudo?
This study is about a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. PNH happens when red blood cells are destroyed too early, which can lead to symptoms like tiredness, shortness of breath, and tummy pain. Currently, there are treatments available that help manage PNH by blocking a part of the immune system. This study is testing a new medicine, crovalimab, to see if it's a good alternative or improvement to treatments already in use.
The main aim of the study is to carefully look at how safe crovalimab is for people with PNH who are already being treated with similar medicines. It will also compare how well crovalimab works against one of the established treatments, called eculizumab. By doing this, researchers hope to understand if crovalimab could become another helpful option for patients.
This kind of research is very important because it helps doctors and patients make informed decisions about treatment. Finding new medicines that are safe and effective can significantly improve the quality of life for people with rare conditions like PNH. This study is a crucial step in potentially broadening the treatment landscape for this condition.
Pontos principais
- Compares a new medicine (crovalimab) to an existing one (eculizumab) for PNH.
- Aims to check the safety and how well crovalimab works.
- For PNH patients already on similar complement inhibitor treatments.
- Involves regular clinic visits and health monitoring.
- A participação é voluntária, e você pode se retirar a qualquer momento.
Quem pode ser elegível?
To join this study, generally, you need to be at least 2 years old and have a confirmed diagnosis of PNH. You should also have been receiving treatment for your PNH, specifically eculizumab or ravulizumab, for at least 3 months before the study starts. Your blood test results (specifically for a protein called Lactate Dehydrogenase) should be within certain limits, and you need to be able to attend all study appointments and follow the instructions given.
There are also some reasons why you might not be able to join. For example, if you've recently had a bone marrow transplant or have certain types of bone marrow disorders, you wouldn't be eligible. The study also cannot include pregnant or breastfeeding individuals, or those planning to become pregnant during or shortly after the study. If you have active Hepatitis B or C, or have recently taken part in another experimental treatment study, that would also prevent you from joining.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Are you at least 2 years old?
- Do you have a confirmed diagnosis of PNH?
- Have you been treated with eculizumab or ravulizumab for at least 3 months?
- Are you able to attend all study appointments?
- Are you not pregnant or breastfeeding, and not planning to become pregnant soon?
O que envolve a participação?
If you decide to take part, you would receive either the new medicine, crovalimab, or the existing medicine, eculizumab. The study team would carefully monitor your health and how you respond to the treatment. This will involve regular visits to the clinic for check-ups, blood tests, and other medical assessments. The total duration of your participation in the study, including the treatment period and follow-up, would be fully explained to you before you agree to join. Women who could become pregnant would also need to agree to use effective contraception during the study and for a period afterwards.
Riscos e benefícios potenciais
Locations (80)
- Carolinas Healthcare SystemCódigo postal verificadoCharlotte, Estados Unidos
- Nationwide Children's HospitalCódigo postal verificadoColumbus, Estados Unidos
- Cliniques Universitaires Saint-LucCódigo postal verificadoBruxelas, Bélgica
- AZ Delta Campus WestlaanCódigo postal verificadoRoeselare, Belgium
- Chronos Pesquisa ClinicaCódigo postal verificadoTaguatinga, Brazil
- Santa Casa de Misericordia de Porto AlegreCódigo postal verificadoPorto Alegre, Brazil
- Hospital de Clínicas de Porto Alegre XCódigo postal verificadoPorto Alegre, Brazil
- Instituto Joinvilense de Hematologia E OncologiaCódigo postal verificadoJoinville, Brazil
- Hospital das Clínicas FMRP-USPCódigo postal verificadoRibeirão Preto, Brazil
- *X*CEPHO - Centro de Estudos e Pesquisas em Hematologia e OncologiaCódigo postal verificadoSanto André, Brazil
- Hospital Sírio-LibanêsCódigo postal verificadoSão Paulo, Brazil
- Hospital PaulistanoCódigo postal verificadoSão Paulo, Brazil
Perguntas frequentes
What is PNH?
PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare blood disorder where red blood cells are destroyed too soon, leading to symptoms like tiredness and anaemia.
What are crovalimab and eculizumab?
Crovalimab is a new medicine being tested, and eculizumab is an existing medicine, both used to treat PNH by targeting the immune system.
Who is this study for?
This study is for people with PNH who are already being treated with similar medicines like eculizumab or ravulizumab.
Will I know which medicine I am getting?
Typically in studies like this, you might not know whether you're receiving the new medicine or the existing one. This helps researchers compare the treatments fairly.
Quanto tempo dura o estudo?
The exact duration of the study, including treatment and follow-up, will be fully explained by the study team before you decide to participate.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.