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Aktiv, aber nicht rekrutierendPHASE3INTERVENTIONELL

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.

Auf einen Blick

Status
Aktiv, aber nicht rekrutierend
Phase
PHASE3
Sponsor
Hoffmann-La Roche
Einschreibeziel
210
Starten
08 Okt 2020
Geschätzte Fertigstellung
18 Apr 2027

Ergebnisse

Ergebnisse dieser Studie

Veröffentlicht November 2025

Die Ergebnisse dieser Studie wurden veröffentlicht.

Hauptausgang
Percentage of Participants With Transfusion Avoidance (TA)
TA is defined as participants who are packed red blood cell (pRBC) transfusion-free and do not require transfusion per protocol-specified guidelines. 95% Confidence Interval (CI) for percentage of participants with TA was calculated using the Wilson's method with continuity correction. Participants who withdrew before Week 25 were deemed to have had a transfusion. Percentages are rounded off to the nearest single decimal.
Vollständige Ergebnisse im Register

Worum geht es in dieser Studie?

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.

Wichtige Erkenntnisse

  • 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.

Wer könnte berechtigt sein?

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.

Könnte diese Studie für Sie geeignet sein?

Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.

  1. I have been diagnosed with paroxysmal nocturnal haemoglobinuria (PNH).
  2. I am at least 2 years old.
  3. I have never received eculizumab or another complement inhibitor before.
  4. I have not had a bone marrow or stem cell transplant.
  5. I am vaccinated against meningitis, or willing to receive the vaccine.
  6. I am not pregnant, not breastfeeding, and willing to use effective birth control if applicable.
Beantworten Sie jede Frage, um Ihr Ergebnis zu sehen.

Was beinhaltet die Teilnahme?

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.

Potenzielle Risiken und Vorteile

Taking part may help manage your PNH symptoms and keep your blood cell levels stable, though this cannot be guaranteed. You will also have regular, detailed health monitoring from blood specialists. However, all medicines carry risks of side effects. Because these treatments pause part of your immune system, they can increase the risk of serious infections, particularly meningitis, which is why vaccinations are required. Taking part is completely your choice, and you can withdraw at any time without your regular NHS care being affected.

Standorte (63)

  • Organizacion Medica de Investigacion (OMI)
    Nur Stadt
    Ciudad Autonoma Buenos Aires, Argentina
  • *X*CEPHO - Centro de Estudos e Pesquisas em Hematologia e Oncologia
    Verifizierte Postleitzahl
    Santo André, Brazil
  • Beneficencia Portuguesa de Sao Paulo
    Verifizierte Postleitzahl
    São Paulo, Brazil
  • Peking Union Medical College Hospital
    Verifizierte Postleitzahl
    Peking, China
  • Nanfang Hospital, Southern Medical University
    Verifizierte Postleitzahl
    Guangzhou, China
  • The First Affiliated Hospital, Zhejiang University
    Verifizierte Postleitzahl
    Hangzhou, China
  • Jiangsu Province Hospital
    Verifizierte Postleitzahl
    Nanjing, China
  • Affiliated Hospital of Nantong University
    Verifizierte Postleitzahl
    Nantong, China
  • Huashan Hospital, Fudan University
    Verifizierte Postleitzahl
    Shanghai, China
  • Union Hospital Tongji Medical College Huazhong University of Science and Technology
    Verifizierte Postleitzahl
    Wuhan, China
  • Centre Hospitalier Lyon Sud
    Unbestätigt
    Pierre-Bnite, France
  • Hopital Claude Huriez - CHU Lille
    Verifizierte Postleitzahl
    Lille, Frankreich

Häufige Fragen

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.

Kann ich die Studie verlassen, wenn ich meine Meinung ändere?

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.

Wie Sie mehr erfahren können

Sprechen Sie immer mit Ihrem Hausarzt oder Spezialisten, bevor Sie sich entscheiden, an einer Studie teilzunehmen.

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