Long-term safety and effectiveness study of pozelimab and cemdisiran combination treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH)

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What is this study about?

This study focuses on patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder where the immune system attacks and destroys red blood cells, causing them to break down prematurely. The study will evaluate a combination treatment using two medications: Pozelimab and Cemdisiran. Both medications are given as solutions for injection under the skin.

The main purpose of this research is to understand how safe and effective this combination therapy is when used for a long time in patients with PNH. The study will look at how well the treatment works by monitoring various aspects of the disease, including the levels of a substance called lactate dehydrogenase (LDH) in the blood, which indicates the extent of red blood cell destruction.

During the study, participants will receive both medications through subcutaneous injections. The treatment period will last for approximately 108 weeks, during which researchers will monitor the patients’ response to the treatment and any side effects that may occur. The study will also track other important measures such as the need for blood transfusions and changes in patients’ quality of life.

1 Initial treatment phase

You will receive two medications: pozelimab and cemdisiran

Both medications will be given as injections under the skin (subcutaneous injection)

The treatment continues as part of a long-term safety and effectiveness study

2 Regular monitoring

Your blood will be tested regularly to measure lactate dehydrogenase (LDH) levels, which indicate how well the treatment is working

Your hemoglobin levels will be monitored throughout the study

Blood samples will be taken to check the levels of both medications in your system

3 Safety assessments

Regular health checks will be performed to monitor for any side effects

You will need to receive meningococcal vaccinations as required by the study protocol

Any unusual symptoms or health changes should be reported during clinic visits

4 Quality of life evaluations

You will complete questionnaires about your fatigue levels

Your physical function will be assessed using specific evaluation tools

Your overall quality of life will be monitored throughout the study

5 Long-term follow-up

The study will continue until May 2029

Regular clinic visits will be required throughout the study period

Your response to treatment will be monitored continuously

Who Can Join the Study?

Must have completed the previous study (parent study) for PNH treatment without permanently stopping the treatment
Must be willing and able to attend clinic visits and follow study procedures, including getting required meningococcal vaccinations (vaccines that protect against certain bacterial infections)
For patients with specific genetic variation: Must have a documented C5 polymorphism (a genetic change that makes certain treatments ineffective)
For patients with specific genetic variation: Must have PNH confirmed by special blood testing called high-sensitivity flow cytometry
For patients with specific genetic variation: Must have active disease with at least one PNH-related sign or symptom
For patients with specific genetic variation: Must have LDH levels (a blood test that measures tissue damage) at least 2 times higher than the normal upper limit during screening
Both male and female patients can participate
Must be an adult or adolescent (study includes people aged 12 years and older)

Who Cannot Join the Study?

History of bone marrow failure syndrome (conditions affecting blood cell production in bone marrow)
Active or chronic bacterial infections
Known or suspected active viral hepatitis (liver inflammation caused by viruses)
Current HIV infection
History of major organ transplantation
Any active malignancy (cancer) or history of cancer within the past 5 years
Severe kidney disease requiring dialysis
Severe liver disease
History of blood clotting disorders
Participation in another clinical trial within the past 30 days
Pregnant or breastfeeding women
Known allergic reactions to similar medications
Significant heart conditions or uncontrolled high blood pressure
Use of certain medications that could interact with the study drug
Mental conditions that could affect the ability to provide informed consent

Where you can join this trial?

Verified and Recommended Sites

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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
Hospital Clinic De Barcelona	Barcelona	Spain
Semmelweis University	Budapest	Hungary
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca	Cluj Napoca	Romania
In Vivo Sp. z o.o.	Bydgoszcz	Poland
Emergency Institute For Cardiovascular Diseases And Transplant	Targu Mures	Romania
Sxbfqptx Cqftio Mjbopzyat Fqhwufcqmaq Cmzvxxo	Craiova	Romania
Axlsijx Odifluiqjfw Updbkcjpwxpkx Cetzbddzkafs Dbjjr Stfvoe E Dbxbh Sxwhfqc Di Tqioqw	Turin	Italy
Uydwainwbkqyda Cffymgd Ktljymnhi	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Greece	Not recruiting	26.03.2023
Hungary	Recruiting	26.03.2023
Italy	Recruiting	26.03.2023
Poland	Recruiting	26.03.2023
Romania	Recruiting	26.03.2023
Spain	Recruiting	26.03.2023

Trial locations

Investigated drugs:

Pozelimab is a medication designed to treat patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. It works by targeting specific proteins in the immune system that are involved in the destruction of red blood cells.

Cemdisiran is also used in treating Paroxysmal Nocturnal Hemoglobinuria (PNH). It works by reducing the activity of certain proteins that contribute to the breakdown of red blood cells in patients with PNH.

The combination of these two medications (Pozelimab and Cemdisiran) is being studied to see how well they work together over a long period and how safe they are when used in combination to treat patients with PNH. This combination therapy approach aims to provide better control of the disease symptoms than using either medication alone.

Investigated diseases:

Paroxysmal nocturnal haemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria (PNH) – A rare blood disorder where the body’s immune system attacks and destroys its own red blood cells, leading to their premature breakdown. The condition occurs due to genetic changes in blood-forming stem cells, causing red blood cells to become sensitive to destruction by the body’s complement system. The disease gets its name from the historical observation that symptoms often worsen at night, though this can occur at any time. Patients experience episodes of red blood cell destruction, which can lead to anemia and the release of hemoglobin in the urine, making it appear dark. The condition is chronic and can affect multiple body systems.

Trial ID:

2023-510336-36-00

Protocol code:

R3918-PNH-2050

NCT ID:

NCT05744921

Trial Phase:

Therapeutic confirmatory (Phase III)

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Long-term safety and effectiveness study of pozelimab and cemdisiran combination treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?