A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare condition that causes the body's immune system to attack and break down its own red blood cells. This can lead to severe tiredness, anaemia, and an increased risk of dangerous blood clots. The current standard treatment is eculizumab, which is given through a drip into a vein every two weeks. This Phase 3 trial is testing a newer medicine called crovalimab to see if it works just as well. Crovalimab targets the same part of the immune system to protect red blood cells. The trial is open to adults and children aged 2 and older with active PNH who have never received this type of targeted treatment before. Participants are closely monitored by a specialist medical team throughout the study. Joining is completely voluntary, and you can leave at any time.
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Risultati
Risultati di questo studio
Pubblicato November 2025I risultati di questo studio sono stati pubblicati.
Di cosa tratta questo studio?
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, long-term blood condition. In people with PNH, part of the immune system called the 'complement system' mistakenly attacks and destroys healthy red blood cells. When these cells break open, it causes problems such as severe fatigue, dark urine, kidney strain, and blood clots.
Currently, many people with PNH are treated with eculizumab. This medicine blocks the complement system to stop blood cells from breaking down, but it requires regular visits to a clinic for infusions into a vein. Researchers have developed crovalimab, a newer medicine that works in a similar way. This study is designed to see whether crovalimab is just as effective and safe as eculizumab for people who are starting treatment for the first time.
Finding new ways to treat PNH is important. If crovalimab works well, it could provide doctors and patients with another reliable choice for managing PNH, potentially offering more convenience and flexibility in daily life while keeping symptoms under control.
Punti chiave
- The trial compares a newer medicine (crovalimab) with the standard treatment (eculizumab) for PNH.
- It is designed for adults and children aged 2 and older who have never used complement inhibitor treatments before.
- A meningitis vaccination is required before treatment to help keep you safe from infection.
- Participants will have regular check-ups, blood tests, and specialist support throughout.
- Taking part is completely voluntary and will not impact your normal NHS treatment rights.
Chi può essere idoneo?
This study is looking for adults and children aged 2 or older who have been diagnosed with PNH. To take part, your blood tests must show signs that red blood cells are actively breaking down (measured by high levels of an enzyme called LDH). You must also have had a vaccination against meningococcal bacteria (a common cause of meningitis) or be willing to receive one before starting the study.
You cannot take part if you have ever been treated with a complement inhibitor medicine before, or if you have had a bone marrow or stem cell transplant. People with certain serious bone marrow disorders, or those who have had a meningitis infection within the last six months, are also not eligible.
Because the effects of these medicines on unborn babies are not fully known, you cannot take part if you are pregnant or breastfeeding. Women who can become pregnant must agree to use reliable contraception during the trial and for several months after their final dose.
Questo studio potrebbe fare al caso tuo?
Rispondi a queste domande rapide per vedere se potresti essere idoneo. Questo è solo una guida — il team di ricerca prende la decisione finale.
- I have been diagnosed with paroxysmal nocturnal haemoglobinuria (PNH).
- I am at least 2 years old.
- I have never received eculizumab or another complement inhibitor before.
- I have not had a bone marrow or stem cell transplant.
- I am vaccinated against meningitis, or willing to receive the vaccine.
- I am not pregnant, not breastfeeding, and willing to use effective birth control if applicable.
Cosa comporta la partecipazione?
If you join the study, you will be placed into a group to receive either crovalimab or eculizumab. You will visit the clinic regularly so the medical team can give you your medication, check your overall health, and run routine blood and urine tests. These checks help the team see how well the medicine is protecting your red blood cells and look out for any side effects. The study lasts for several months, and the research team will explain the exact schedule of visits and follow-up checks before you decide to take part.
Rischi e benefici potenziali
Locations (63)
- Organizacion Medica de Investigacion (OMI)Solo cittàCiudad Autonoma Buenos Aires, Argentina
- *X*CEPHO - Centro de Estudos e Pesquisas em Hematologia e OncologiaCodice postale verificatoSanto André, Brazil
- Beneficencia Portuguesa de Sao PauloCodice postale verificatoSão Paulo, Brazil
- Peking Union Medical College HospitalCodice postale verificatoPechino, Cina
- Nanfang Hospital, Southern Medical UniversityCodice postale verificatoGuangzhou, China
- The First Affiliated Hospital, Zhejiang UniversityCodice postale verificatoHangzhou, China
- Jiangsu Province HospitalCodice postale verificatoNanjing, China
- Affiliated Hospital of Nantong UniversityCodice postale verificatoNantong, China
- Huashan Hospital, Fudan UniversityCodice postale verificatoShanghai, China
- Union Hospital Tongji Medical College Huazhong University of Science and TechnologyCodice postale verificatoWuhan, Cina
- Centre Hospitalier Lyon SudNon verificatoPierre-Bnite, France
- Hopital Claude Huriez - CHU LilleCodice postale verificatoLille, Francia
Domande comuni
What is the main difference between the two study drugs?
Both crovalimab and eculizumab block the part of the immune system that attacks red blood cells. Eculizumab is the standard medicine given through a drip, while crovalimab is a newer medicine designed to work in a similar way with potentially different dosing options.
Why do I need a meningitis vaccine to join?
Both medicines lower your body's defences against specific bacteria that can cause meningitis. Getting vaccinated before starting treatment helps protect you from serious infections.
Will I have to pay to take part in this trial?
No. The trial medication, tests, and study visits are provided free of charge, and trial sponsors often help cover reasonable travel expenses to the hospital.
Posso abbandonare lo studio se cambio idea?
Yes. Taking part is completely voluntary. You are free to stop at any time, and doing so will not affect your standard NHS medical care.
Can children join this study?
Yes, children aged 2 and older may be eligible, provided they meet the specific health, weight, and safety requirements of the study.
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