Study on the Safety and Effectiveness of Crovalimab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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What is this study about?

This clinical trial is focused on studying a condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a rare blood disease that causes red blood cells to break down too early, leading to various health issues. The study is testing a treatment called Crovalimab, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins made in a lab that can bind to substances in the body, such as harmful cells, and help the immune system destroy them. Crovalimab is designed to block a part of the immune system that is overactive in people with PNH.

The purpose of the study is to evaluate the safety and effectiveness of Crovalimab in both healthy volunteers and patients with PNH. The study will be conducted in several parts. Initially, healthy volunteers will receive a single dose of Crovalimab to assess its safety. Following this, patients with PNH who have not been treated with similar medications before, as well as those switching from another treatment, will receive multiple doses over a period of five months. The study will also include an open-label extension phase to assess the long-term safety of Crovalimab.

Participants in the study will receive Crovalimab through injections or infusions, which are methods of delivering medication directly into the body. The study will monitor how the body processes the medication and its effects on the disease. This includes measuring changes in blood markers and symptoms related to PNH, such as fatigue and quality of life. The study aims to provide valuable information on how Crovalimab can help manage PNH and improve the lives of those affected by this condition.

1 initial visit and screening

Upon joining the study, you will attend an initial visit where medical history and current health status are reviewed. This includes a physical examination and blood tests to ensure eligibility for the trial.

Vaccinations against Neisseria meningitidis are required, following local guidelines. If not already vaccinated, this will be arranged during the screening process.

2 treatment initiation

If eligible, you will begin treatment with crovalimab. This medication is administered either as a subcutaneous injection or an intravenous infusion, depending on the specific requirements of the study part you are enrolled in.

The initial dose will be given under medical supervision to monitor for any immediate reactions.

3 regular treatment and monitoring

You will receive regular doses of crovalimab over a period of 5 months. The frequency and dosage will be determined by the study protocol and your specific needs.

Regular follow-up visits will be scheduled to monitor your response to the treatment and to check for any side effects. Blood tests and other assessments will be conducted during these visits.

4 open label extension (optional)

If you complete the initial treatment phase and derive benefit from crovalimab, you may be eligible to continue in an open label extension phase. This allows for long-term monitoring and continued access to the medication.

During this phase, regular assessments will continue to ensure ongoing safety and effectiveness of the treatment.

5 end of study

At the end of your participation in the study, a final visit will be conducted. This includes a comprehensive health assessment and discussion of your experience during the trial.

You will receive guidance on any further medical care or follow-up that may be necessary after the study concludes.

Who Can Join the Study?

For Part 1 (Healthy Volunteers):
- Must be a healthy male between 21 and 55 years old.
- Must have received a vaccination against Neisseria meningitidis, which is a type of bacteria that can cause serious infections, covering serogroups B, A, C, W, and Y.
- Must test negative for hepatitis B, hepatitis C, and HIV.
- Must have been vaccinated against hepatitis B.
- Must agree not to smoke during the in-clinic part of the study.
For Parts 2, 3, and 4 (Patients with PNH):
- Must be a male or female between 18 and 75 years old.
- Must have received a vaccination against Neisseria meningitidis according to local guidelines or standard care for those at increased risk.
- Must have a stable dose of other therapies like immunosuppressants, corticosteroids, or iron supplements for at least 28 days before screening.
- Women who can have children must have a negative pregnancy test.
For Part 2 and Part 4 Arm A (Untreated PNH Patients):
- Hepatitis B patients can join if their liver function tests are less than 2 times the upper limit of normal and there is no liver function impairment.
- Must not have been treated with any complement inhibitor or stopped treatment due to lack of efficacy based on a specific genetic mutation.
For Part 3 and Part 4 Arm B (PNH Patients Treated with Eculizumab):
- Must test negative for hepatitis B, hepatitis C, and HIV, but those treated successfully for hepatitis C or B are eligible.
- Must be adequately controlled in the opinion of the investigator.
- Must receive regular infusions of eculizumab, a medication used to treat PNH.
For Open Label Extension (OLE) (PNH Patients):
- Must have completed Parts 2, 3, and 4 of the study.
- Must have benefited from treatment with crovalimab, according to the investigator.
- Women who are not post-menopausal or have not had surgical sterilization must agree to use effective contraception or remain abstinent during treatment and for a specified period after the last dose.
- Must maintain current vaccinations for Neisseria meningitidis serotypes A, C, W, Y, and B according to local guidelines or standard care.

Who Cannot Join the Study?

Patients with any other serious medical conditions that might affect their participation.
Patients who have had an allergic reaction to similar medications in the past.
Patients who are currently participating in another clinical trial.
Patients who are pregnant or breastfeeding.
Patients with a history of drug or alcohol abuse.
Patients who have received any other investigational drug within the last 30 days.
Patients with a history of certain blood disorders.
Patients who have had a major surgery within the last 3 months.
Patients with uncontrolled high blood pressure.
Patients with active infections that require treatment.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy

Other Sites

Site Name	City	Country
Universitaetsklinikum Aachen AöR	Aachen	Germany
Centre Hospitalier Lyon Sud	Pierre Benite	France
Azienda Unita Locale Socio Sanitaria N 8 Berica	Vicenza	Italy
Somogy Varmegyei Kaposi Mor Oktato Korhaz	Kaposvar	Hungary
Semmelweis University	Budapest	Hungary
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH	Ulm	Germany
Uyljsxexolrjghbewrgwc Etywp Aaf	Essen	Germany
Ahqnixojkf Pvongxrx Hrhoqwfm Db Pasij	Paris	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	30.09.2016
Germany	Not recruiting	30.09.2016
Hungary	Not recruiting	30.09.2016
Italy	Not recruiting	30.09.2016

Trial locations

Investigated drugs:

Crovalimab

Crovalimab is a medication being studied for its potential to help people with a condition called paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare blood disorder that can cause red blood cells to break down too early, leading to various health problems. Crovalimab works by targeting a part of the immune system known as the complement system, which is involved in this breakdown of red blood cells. The study aims to see if crovalimab is safe and how well it works in both healthy volunteers and patients with PNH. It also looks at how the body processes the medication and its effects over time. The goal is to find out if crovalimab can be a safe and effective treatment option for people with PNH.

Paroxysmal nocturnal hemoglobinuria (PNH) – Paroxysmal nocturnal hemoglobinuria is a rare blood disorder characterized by the destruction of red blood cells. This destruction occurs due to a defect in the surface proteins of red blood cells, making them susceptible to attack by the body’s immune system. The disease often leads to episodes of dark-colored urine, especially noticeable in the morning. Over time, the breakdown of red blood cells can cause anemia, leading to symptoms such as fatigue and weakness. Patients may also experience blood clots and impaired bone marrow function. The progression of PNH can vary, with some individuals experiencing mild symptoms and others facing more severe complications.

Trial ID:

2023-506287-14-00

Protocol code:

BP39144

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on the Safety and Effectiveness of Crovalimab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

What is this study about?

Who Can Join the Study?

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