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Study Comparing Crovalimab and Eculizumab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently on Complement Inhibitors

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What is this study about?

This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is comparing two treatments: Crovalimab and Eculizumab. Both are medications that help manage PNH by inhibiting a part of the immune system known as the complement system, which is overactive in people with PNH.

The purpose of the study is to evaluate the safety and tolerability of Crovalimab compared to Eculizumab. Participants in the study will receive either Crovalimab or Eculizumab through injections or infusions. The study will monitor participants over a period to observe any side effects and how well the treatments work in managing the symptoms of PNH. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the actual treatments.

Throughout the study, participants will have regular check-ups to assess their health and the effectiveness of the treatment. This includes monitoring vital signs, blood tests, and any reactions to the medication. The study aims to provide valuable information on how these treatments can help people with PNH and improve their quality of life. The trial is expected to continue until 2030, allowing for comprehensive data collection and analysis.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying a documented diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) and ensuring a body weight of at least 40 kg.

A blood test is performed to check platelet count and lactate dehydrogenase (LDH) levels. Platelet count must be at least 30,000/mm³ without recent transfusion support, and LDH levels should be 1.5 times the upper limit of normal or lower.

2 randomization and treatment allocation

Participants are randomly assigned to one of the study groups. Those in the randomized arms (Arm A and B) must be at least 18 years old and have completed a minimum of 24 weeks of treatment with eculizumab prior to the study.

Participants in the non-randomized arm (Arm C) include those under 18 years old or those with specific treatment histories or conditions, such as treatment with ravulizumab or higher-than-approved doses of eculizumab.

3 treatment administration

Participants receive either crovalimab or eculizumab. Crovalimab can be administered intravenously (IV) or subcutaneously (SC), while eculizumab is administered intravenously.

The frequency and dosage of the medication depend on the specific treatment group and individual patient needs.

4 monitoring and follow-up

Regular monitoring is conducted to assess the safety and effectiveness of the treatment. This includes tracking any adverse events, changes in vital signs, and laboratory test results.

Participants are observed for any reactions at the injection site, infusion-related reactions, and signs of hypersensitivity or infection.

5 evaluation of treatment outcomes

The study evaluates the concentration of crovalimab or eculizumab in the blood, the presence of anti-drug antibodies, and changes in biomarkers related to the disease.

The effectiveness of the treatment is assessed by measuring changes in hemolysis parameters, LDH levels, and hemoglobin stabilization over a 25-week period.

6 completion of study participation

At the end of the study period, participants complete questionnaires to assess treatment satisfaction and preference.

The study is estimated to conclude by January 31, 2030, with ongoing assessments throughout the participation period.

Who Can Join the Study?

  • Must weigh at least 40 kg (about 88 pounds) at the start of the study.
  • Must have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), which is a rare blood condition, confirmed by a special test called high sensitivity flow cytometry of white blood cells (WBCs).
  • Must have a platelet count of at least 30,000 per cubic millimeter at the start of the study, without needing a blood transfusion within 7 days before the test. Platelets are small blood cells that help with clotting.
  • For patients in certain study groups (Arm A and B), must be 18 years or older and have been treated with a medication called eculizumab for at least 24 weeks before the study starts.
  • For patients in certain study groups (Arm A and B), must have a level of a substance called lactate dehydrogenase (LDH) that is no more than 1.5 times the upper limit of normal at the start of the study. LDH is an enzyme that can be measured in the blood.
  • For patients in another study group (Arm C), must be under 18 years old and currently treated with eculizumab, or currently treated with another medication called ravulizumab, or treated with higher-than-approved doses of eculizumab, or have a known C5 polymorphism with poorly controlled hemolysis by eculizumab or ravulizumab. Hemolysis is the breakdown of red blood cells.
  • Note: In France and the Czech Republic, patients under 18 years of age are not eligible to participate in the study.

Who Cannot Join the Study?

  • Patients with a history of severe allergic reactions to any of the study medications.
  • Patients who have received another investigational drug within the last 30 days.
  • Patients with active infections that require treatment with antibiotics.
  • Patients with a history of certain types of cancer.
  • Patients who are pregnant or breastfeeding.
  • Patients with a history of drug or alcohol abuse.
  • Patients with certain heart conditions.
  • Patients who have had a major surgery within the last 3 months.
  • Patients with uncontrolled high blood pressure.
  • Patients with severe liver or kidney disease.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Careggi Florence Italy
Hospital Universitario De Salamanca Salamanca Spain
Hospital Clinico San Carlos Madrid Spain
Centre Hospitalier Universitaire De Lille Lille France

Other Sites

Site Name City Country Status
Universitaetsklinikum Aachen AöR Aachen Germany
Institute Of Hematology And Blood Transfusion Prague Czechia
Hospital General Universitario Gregorio Maranon Madrid Spain
Algemeen Ziekenhuis Delta Roeselare Belgium
Geniko Nosokomeio Thessalonikis George Papanikolaou Thessaloniki Greece
Mtz Clinical Research Powered By Pratia Warsaw Poland
Hospital Universitario Basurto Bilbao Spain
El Hospital Universitario De Gran Canaria Dr. Negrin Las Palmas De Gran Canaria Spain
Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy Bydgoszcz Poland
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Universidade De Santiago De Compostela Santiago De Compostela Spain
ELBLANDKLINIKEN Stiftung & Co. KG Riesa Germany
Samodzielny Publiczny Szpital Kliniczny Nr 1 W Lublinie Lublin Poland
Centro Hospitalar Do Baixo Vouga E.P.E. (CHBV E.P.E.) Aveiro Portugal
Centre Hospitalier Universitaire De Rennes Rennes France
Centro Hospitalar Universitario De Santo Antonio E.P.E. Porto Portugal
Azienda Unita Sanitaria Locale Della Romagna Faenza Italy
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur Namur Belgium
North Estonia Medical Centre Foundation Tallin Estonia
Cevmammps Uscbsvyczcnldz Sfwtoywsa Woluwe-Saint-Lambert Belgium
Ltwyp Gkipiec Hqplpmxq Oc Auwvrh Athens Greece
Acnlljioi Udh Amsterdam The Netherlands
Ubrxofm Ukzdbwqkst Hcrocysu Uppsala Sweden
Auoanuw Oxzrocpmaoo Useaabkgdpyht Cwzgnzpdfalo Dadmx Sjtmxr E Dycqg Sfabqkc Dp Txfzgq Turin Italy
Ueviitujueffkt Cihirfr Kqpztqyvh Gdansk Poland
Ikxcrqau Petoseqhtqzpozl Cxompj Coklmc Marseille France
Urjdlbumgz Goqsjzs Hingnqhn Awbzsdx Athens Greece

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
31.08.2020
Czechia Czechia
Not recruiting
31.08.2020
Estonia Estonia
Not recruiting
31.08.2020
France France
Not recruiting
31.08.2020
Germany Germany
Not recruiting
31.08.2020
Greece Greece
Not recruiting
31.08.2020
Ireland Ireland
Not recruiting
31.08.2020
Italy Italy
Not recruiting
31.08.2020
Poland Poland
Not recruiting
31.08.2020
Portugal Portugal
Not recruiting
31.08.2020
Spain Spain
Not recruiting
31.08.2020
Sweden Sweden
Not recruiting
31.08.2020
The Netherlands The Netherlands
Not recruiting
31.08.2020

Trial locations

Investigated drugs:

Crovalimab is a medication being studied for its potential to treat patients with a condition called paroxysmal nocturnal hemoglobinuria (PNH). This condition affects the blood, and crovalimab is designed to help by inhibiting certain proteins in the blood that can cause damage to red blood cells. The trial aims to see how safe and effective crovalimab is for patients who are already receiving treatment with other similar medications.

Eculizumab is another medication used in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). It works by blocking specific proteins in the blood that can lead to the destruction of red blood cells. In this trial, eculizumab is used as a comparison to evaluate the safety and effectiveness of crovalimab. Patients in the trial are already being treated with eculizumab, and the study will compare the outcomes of those continuing with eculizumab to those switching to crovalimab.

Investigated diseases:

Paroxysmal Nocturnal Hemoglobinuria (PNH) – This is a rare blood disorder characterized by the destruction of red blood cells, leading to symptoms such as fatigue, shortness of breath, and dark-colored urine. The disease occurs due to a mutation in the PIGA gene, which affects the surface proteins that protect red blood cells from the immune system. Over time, the destruction of red blood cells can lead to anemia and other complications. Patients may experience episodes of hemolysis, where red blood cells break down more rapidly, often at night. The condition can also cause blood clots, which may lead to further health issues. PNH is a chronic condition that requires ongoing management to address its symptoms and complications.

Trial ID:
2023-506526-37-00
Protocol code:
BO42161
Trial Phase:
Therapeutic confirmatory (Phase III)

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