Study on the Safety and Effectiveness of Pozelimab and Cemdisiran for Adults with Paroxysmal Nocturnal Hemoglobinuria Not Recently 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 testing a combination of two treatments, Pozelimab and Cemdisiran, to see how safe and effective they are for patients with PNH who have not recently received or have never received treatment with complement inhibitors, which are drugs that help prevent the breakdown of red blood cells.

The purpose of the study is to compare the effects of the combination therapy of Pozelimab and Cemdisiran with other treatments, such as Ravulizumab and Eculizumab, which are also used to treat PNH. The study will last for 26 weeks, during which participants will receive either the combination therapy or one of the other treatments. The study will monitor how well the treatments control the breakdown of red blood cells and whether they help avoid the need for blood transfusions.

Participants will be divided into two groups, known as cohorts. One group will receive the combination therapy and be compared to those receiving Ravulizumab, while the other group will be compared to those receiving Eculizumab. The study will assess the effects of the treatments by measuring levels of a substance called lactate dehydrogenase (LDH) in the blood, which indicates how much red blood cell breakdown is occurring. The study aims to provide valuable information on the safety and effectiveness of the new combination therapy for managing PNH.

1 initial visit

Upon joining the study, you will attend an initial visit. During this visit, your eligibility will be confirmed through tests, including high-sensitivity flow cytometry to check for paroxysmal nocturnal hemoglobinuria (PNH).

You will be informed about the study procedures and asked to provide consent to participate. This visit will also include a review of your medical history and a physical examination.

2 treatment assignment

You will be randomly assigned to one of two groups. One group will receive a combination of pozelimab and cemdisiran, while the other group will receive either ravulizumab or eculizumab.

The assignment is random to ensure the study’s scientific validity.

3 treatment administration

If you are in the pozelimab and cemdisiran group, you will receive these medications as a solution for injection. The frequency and dosage will be explained to you by the study team.

If you are in the ravulizumab or eculizumab group, you will receive these medications as a solution for infusion. The study team will provide details on the dosage and frequency.

4 regular monitoring

Throughout the 26-week treatment period, you will have regular clinic visits to monitor your health and the effects of the treatment.

These visits will include blood tests to measure lactate dehydrogenase (LDH) levels, which help assess the treatment’s impact on hemolysis, a process where red blood cells break down.

5 follow-up visits

After completing the treatment period, you will have follow-up visits to ensure your well-being and to gather additional data on the treatment’s long-term effects.

These visits will include assessments similar to those during the treatment period, such as blood tests and health evaluations.

Who Can Join the Study?

Must have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) through a special blood test called high-sensitivity flow cytometry.
Must have active PNH, which means experiencing one or more signs or symptoms related to PNH.
Must have a level of lactate dehydrogenase (LDH) that is at least twice the upper limit of normal (ULN) at the screening visit. LDH is an enzyme that can indicate cell damage when elevated.
Must be willing and able to attend clinic or remote visits and follow study-related procedures, including completing all required meningococcal vaccinations. These are vaccines to protect against certain types of bacterial infections.
Open to both male and female participants.
Participants must be within the specified age range for the study.

Who Cannot Join the Study?

Patients who have received a complement inhibitor treatment recently cannot participate. A complement inhibitor is a type of medication used to treat certain blood disorders.
Patients who are not treatment-naive for complement inhibitors are excluded. Treatment-naive means they have not been treated with this type of medication before.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Hospital Universitario De Salamanca	Salamanca	Spain

Other Sites

Site Name	City	Country
Hospital General Universitario Morales Meseguer	Murcia	Spain
Instytut Hematologii I Transfuzjologii	Warsaw	Poland
Geniko Nosokomeio Thessalonikis George Papanikolaou	Thessaloniki	Greece
Hospital Universitario Basurto	Bilbao	Spain
Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy	Bydgoszcz	Poland
Hospital Clinic De Barcelona	Barcelona	Spain
Semmelweis University	Budapest	Hungary
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca	Cluj Napoca	Romania
Emergency Institute For Cardiovascular Diseases And Transplant	Targu Mures	Romania
Seiaehtb Cccwwi Mfuzrtjap Fyidvzxkcbl Cgcljhf	Craiova	Romania
Apishyy Osxvzyasfuu Uoodirhgogzov Cwrlcfsuyghy Dwfte Slmqgg E Dfxun Srteetg Dt Tsbwul	Turin	Italy
Ubdmogorqouiea Cnxxvmf Kkuvxyrbm	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Greece	Not recruiting	15.09.2022
Hungary	Not recruiting	15.09.2022
Italy	Not recruiting	15.09.2022
Poland	Not recruiting	15.09.2022
Romania	Not recruiting	15.09.2022
Spain	Not recruiting	15.09.2022

Trial locations

Investigated drugs:

Pozelimab is a medication being studied for its ability to help patients with a condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a disease where red blood cells break down too easily, leading to various health problems. Pozelimab works by blocking a part of the immune system that is overactive in PNH, which can help reduce the breakdown of red blood cells.

Cemdisiran is another medication being tested in combination with Pozelimab for treating PNH. Like Pozelimab, Cemdisiran aims to control the overactive part of the immune system that causes red blood cells to break down. By using both medications together, the study hopes to see a better effect in managing the symptoms of PNH.

Ravulizumab is a medication already used to treat PNH. It helps prevent the breakdown of red blood cells by blocking the same part of the immune system that Pozelimab and Cemdisiran target. In this study, Ravulizumab is used as a comparison to see how well the new combination of Pozelimab and Cemdisiran works.

Eculizumab is another existing treatment for PNH. It also works by stopping the immune system from breaking down red blood cells too quickly. In the trial, Eculizumab is used to compare its effectiveness with the new combination of Pozelimab and Cemdisiran, to see if the new treatment offers any advantages.

Investigated diseases:

Paroxysmal nocturnal haemoglobinuria

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 vulnerable to attack by the body’s immune system. The disease often leads to episodes of dark-colored urine, especially noticeable in the morning, due to the release of hemoglobin from destroyed red blood cells. Over time, PNH can cause fatigue, shortness of breath, and an increased risk of blood clots. The progression of the disease can vary, with some individuals experiencing mild symptoms and others facing more severe complications. The condition is chronic and requires ongoing monitoring to manage its effects on the body.

Trial ID:

2023-509657-31-00

Protocol code:

R3918-PNH-2021

NCT ID:

NCT05133531

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effectiveness of Pozelimab and Cemdisiran for Adults with Paroxysmal Nocturnal Hemoglobinuria Not Recently Treated with Complement Inhibitors

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?