Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

This research study is investigating a new combination of two medications, pozelimab and cemdisiran, for adults living with a rare blood disorder called Paroxysmal Nocturnal Hemoglobinuria (PNH). The main goals are to find out how safe this new combination is and how well it works. We're also comparing its effects to two established PNH treatments, ravulizumab and eculizumab. Participants are adults who haven't recently received or have never had treatment with complement inhibitor medicines for their PNH. The study will help us understand side effects, how the body handles these new drugs, and whether the body might make antibodies against them, which could affect their usefulness.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Regeneron Pharmaceuticals

Enrolment target

202

Start

01 Aug 2022

Estimated completion

26 Oct 2026

Paroxysmal Nocturnal Hemoglobinuria

What is this study about?

This study is exploring potential new treatments for a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, often shortened to PNH. PNH causes red blood cells to break down too early, leading to symptoms like tiredness, anaemia, and sometimes blood clots. Currently, there are treatments available, but researchers are always looking for new and potentially better options.

This particular study is focusing on a new combination of two experimental drugs called pozelimab and cemdisiran. The main purpose is to see how safe this combination is for people with PNH and how effectively it helps manage their condition. To understand its benefits, the study will compare this new combination with two existing and commonly used PNH treatments: ravulizumab and eculizumab.

By comparing these treatments, the study aims to answer important questions. We want to know if the new pozelimab and cemdisiran combination is as good as, or even better than, the current treatments. Researchers will also be carefully monitoring for any side effects and learning how the body processes these new medications. This helps us understand if the new treatment could be a valuable option for future PNH patients.

Key takeaways

This study investigates a new combination treatment for PNH.
It compares new drugs (pozelimab + cemdisiran) with existing PNH treatments.
Safety and effectiveness for adults with PNH are key goals.
Participation involves regular clinic visits and health checks.
Meningitis vaccinations are a crucial eligibility requirement.

Who may be eligible?

To join this study, you must be an adult (18 years or older) with PNH that has been officially diagnosed through special blood tests. Your PNH needs to be 'active,' meaning you're currently experiencing symptoms or signs of the condition, and certain blood levels (LDH level) must be above a specific point.

You also need to be able to attend all the study appointments, whether they are in person or online. It's very important that you are willing and able to get specific meningitis vaccinations, if you haven't had them already, as required by the study plan.

There are also reasons why you might not be able to join. For instance, if you've recently had certain PNH treatments (like eculizumab within the last 3 months, or ravulizumab within the last 6 months), or if you've had an organ or bone marrow transplant. You also can't participate if you weigh less than 40 kilograms, plan to use other PNH treatments not part of the study, or have certain infections.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult (18 years or older)?
Have you been diagnosed with PNH?
Are you experiencing active PNH symptoms or signs?
Are you willing and able to get meningitis vaccinations if needed?
Have you not recently received specific PNH complement inhibitor treatments (e.g., eculizumab in last 3 months, ravulizumab in last 6 months)?
Have you NOT had an organ or bone marrow transplant?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will likely receive either the new drug combination (pozelimab + cemdisiran) or one of the existing PNH treatments (ravulizumab or eculizumab). These will be given over a period of time as determined by the study. You will have regular visits, which might be in person at a clinic or sometimes done remotely. During these visits, the study team will carry out health checks, take blood samples to see how the treatment is working, and ask about any side effects you might be experiencing. They will also look at how much medication is in your blood and if your body is making antibodies against the study drugs. The total duration of your participation in the study, including follow-up, will be explained in detail by the study team.

Potential risks and benefits

Taking part in this study could offer some potential benefits, such as receiving a PNH treatment under close medical supervision, potentially including a new investigational drug that might improve your condition. However, there are also potential risks, including side effects from the study drugs, which could be mild or severe. Like any medical treatment, there's no guarantee the new drugs will work for you. You are free to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (68)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The Oncology Institute of Hope & Innovation
Verified postcode
Whittier, United States
Centro de Estudos e Pesquisas em Hematologia e Oncologia
Verified postcode
Santo André, Brazil
A Beneficencia Portuguesa de Sao Paulo, BP Mirante
Verified postcode
São Paulo, Brazil
Casa de Saude Santa Marcelina
Verified postcode
São Paulo, Brazil
Toronto General Hospital
Verified postcode
Toronto, Canada
Peking Union Medical College Hospital
Verified postcode
Beijing, China
Hospital Pablo Tobon Uribe
Verified postcode
Medellín, Colombia
George Papanikolaou Hospital
Verified postcode
Thessaloniki, Greece
Semmelweis University
Verified postcode
Budapest, Hungary
Malabar Cancer Center, Kerala
Verified postcode
Kannur, India
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Verified postcode
Kochi, India
K J Somaiya Super Specialty Hospital & Research Centre
Verified postcode
Mumbai, India

Common questions

What is PNH?

PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare blood condition where red blood cells break down too quickly, which can cause symptoms like tiredness.

Are the new drugs safe?

The study aims to find out how safe the new drug combination, pozelimab and cemdisiran, is. All study medications are carefully monitored for any side effects.

Will I get the new treatment or an existing one?

During the study, you will be given either the new combination treatment or one of two existing, approved treatments for PNH (ravulizumab or eculizumab).

How long will I be in the study?

The exact length of your participation will be fully explained by the study team. It will involve regular visits and check-ups over a period of time.

Can I stop participating at any time?

Yes, you have the right to leave the study at any point, for any reason, without it affecting your medical care.