Study on the Effectiveness and Safety of Crovalimab vs. Eculizumab for Adults and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Treated with 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. Crovalimab is a solution that can be given either through an injection under the skin or directly into a vein, while Eculizumab is given through a vein. Both treatments are designed to help manage the symptoms of PNH by preventing the breakdown of red blood cells.

The purpose of this study is to evaluate how effective and safe Crovalimab is compared to Eculizumab in patients who have not been treated with similar medications before. Participants in the study will receive either Crovalimab or Eculizumab and will be monitored over a period of time to see how well the treatments work in preventing blood transfusions and controlling the breakdown of red blood cells. The study will also look at other factors such as the stability of hemoglobin levels, which is a protein in red blood cells that carries oxygen, and any side effects that may occur.

Throughout the study, researchers will collect information on how patients respond to the treatments, including any changes in fatigue levels, which is a common symptom of PNH. They will also monitor for any reactions at the injection site or other side effects. The study aims to provide valuable insights into the management of PNH and help determine the best treatment options for patients with this condition.

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) through specific blood tests.

Additional criteria include a body weight of at least 40 kg, specific blood cell counts, and agreement to use contraception if applicable.

2 randomization

Participants are randomly assigned to receive either crovalimab or eculizumab. This process ensures that the study results are unbiased.

3 treatment administration

The treatment involves receiving the assigned medication. Crovalimab can be administered either intravenously (IV) or subcutaneously (SC), while eculizumab is administered intravenously.

The frequency and dosage of the medication are determined by the study protocol and are monitored by the study team.

4 monitoring and assessments

Regular monitoring is conducted to assess the effectiveness and safety of the treatment. This includes measuring blood levels of specific markers and evaluating the control of hemolysis.

Participants are also monitored for any side effects or adverse reactions to the medication.

5 evaluation of outcomes

The study evaluates several outcomes, including the ability to avoid blood transfusions and control hemolysis, as well as the impact on fatigue and other health indicators.

The presence of any adverse events or reactions is also recorded and analyzed.

6 completion of study

The study is expected to conclude by June 2028. At the end of the study, a final assessment is conducted to evaluate the overall effectiveness and safety of the treatments.

Who Can Join the Study?

  • Must have a body weight of at least 40 kg (about 88 pounds).
  • 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 level of LDH (Lactate Dehydrogenase, an enzyme found in the blood) that is at least twice the upper limit of normal (ULN) during screening.
  • Must have a platelet count of at least 30,000 per cubic millimeter (mm3) at screening, without needing a blood transfusion within 7 days before the test. Platelets are cells that help with blood clotting.
  • Must have an ANC (Absolute Neutrophil Count, a type of white blood cell) greater than 500 per microliter (micro L) at screening.
  • For female patients who can have children: Must agree to either not have sexual intercourse or use birth control methods to prevent pregnancy.

Who Cannot Join the Study?

  • Patients who have a different condition than Paroxysmal Nocturnal Hemoglobinuria (PNH) cannot participate. PNH is a rare blood disorder.
  • Patients who are not within the specified age range for the study cannot participate. The study includes certain age groups.
  • Patients who are not male or female cannot participate, as the study includes both genders.
  • Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups that might need special protection.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Clinico San Carlos Madrid Spain
Centre Hospitalier Universitaire De Lille Lille France

Other Sites

Site Name City Country Status
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital San Pedro De Alcantara Caceres Spain
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
Hospital Clinic De Barcelona Barcelona Spain
Vilniaus universiteto ligonine Santaros klinikos VšĮ Vilnius Lithuania
Instituto Portugues De Oncologia De Lisboa Francisco Gentil E.P.E. Lisbon Portugal
Spitalul Universitar De Urgenta Bucuresti Bucharest Romania
Samodzielny Publiczny Szpital Kliniczny Nr 1 W Lublinie Lublin Poland
Centro Hospitalar Do Baixo Vouga E.P.E. (CHBV E.P.E.) Aveiro Portugal
Centro Hospitalar Universitario De Santo Antonio E.P.E. Porto Portugal
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Centre Hospitalier Lyon Sud Pierre Benite France
Uawlzttebltorelpnfezt Emgpm Aqq Essen Germany
Unnxqll Ueaaozbxbr Hybrkhby Uppsala Sweden
Avoxilipk Uks Amsterdam The Netherlands
Upfrxxucsvvlzv Cipqube Knptylted Gdansk Poland
Ftplmqxak Pfqv Lq Iobhnrseamqse Bwzbsyevu Dcm Hfyusfxw Uvvppdowzbjpk Lt Pcm Madrid Spain
Hnqmhrxj Uisiabobpwmye Hbhwbfwj Tctbg y Poyrjt Ipatzvjn Cxmrri deaxgijfcjexyignk (gqre Badalona Spain

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

Trial status

Country Status Recruitment Start
France France
Not recruiting
31.08.2020
Germany Germany
Not recruiting
31.08.2020
Lithuania Lithuania
Not recruiting
31.08.2020
Poland Poland
Not recruiting
31.08.2020
Portugal Portugal
Not recruiting
31.08.2020
Romania Romania
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 effectiveness in treating patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). It is designed to work by inhibiting a part of the immune system known as the complement system, which is overactive in PNH patients. This overactivity can lead to the destruction of red blood cells, so crovalimab aims to prevent this process and improve patient outcomes.

Eculizumab is another medication used in the study as a comparison to crovalimab. It is already approved for the treatment of PNH and works similarly by blocking the complement system to prevent the breakdown of red blood cells. The study aims to compare the effectiveness and safety of crovalimab against eculizumab in patients who have not been previously treated with complement inhibitors.

Paroxysmal Nocturnal Hemoglobinuria (PNH) – This is a rare blood disorder where red blood cells break down earlier than normal. It is caused by a mutation in the PIGA gene, leading to a deficiency of proteins that protect red blood cells from the immune system. As a result, patients may experience episodes of dark-colored urine, especially in the morning, due to the release of hemoglobin. Over time, this can lead to symptoms such as fatigue, shortness of breath, and an increased risk of blood clots. The disease can progress with varying severity, and symptoms may fluctuate over time. It primarily affects adults and can significantly impact quality of life.

Trial ID:
2023-506498-36-00
Protocol code:
BO42162
Trial Phase:
Therapeutic confirmatory (Phase III)

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