The ACCESS-1 Study

A clinical study for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are new to treatment* or have not recently received treatment.

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*Treatment with a complement inhibitor

What is the purpose of the ACCESS-1 Study?

The purpose of this study is to learn more about the effects of an experimental combination treatment to see how well-tolerated and effective it is compared to the already approved treatment, eculizumab. Eculizumab is also known as a “complement inhibitor.”*

*Please note that this clinical trial will use investigational drug products, the safety and efficacy of which have not been fully evaluated by Regulatory Authorities and which have not received marketing authorization in any country.

What are the investigational study drugs?

The investigational study drugs work together to reduce the levels and activity of C5, a protein involved in the destruction of red blood cells in people with PNH.

Investigational Study Drug A

An investigational drug called a monoclonal antibody designed to block the activity of C5. After a single infusion (first dose), Investigational Study Drug A is given as an injection under the skin.

Investigational Study Drug B

An investigational drug that reduces the production of C5 protein in the liver. As a result, less total C5 is made. Investigational Study Drug B is given as an injection under the skin.

Eculizumab

Eculizumab is an approved treatment for PNH in some countries. Eculizumab is a monoclonal antibody designed to block the activity of C5 and is given by regular infusions into a vein.

Who can participate in this study?

This study is for adults 18 years of age and older who:

Have a confirmed diagnosis of PNH
Have active disease (≥1 sign or symptom of PNH or history of blood transfusion due to PNH within the last 3 months)
Have not been treated with a complement inhibitor (for example, ravulizumab or eculizumab)

Note: Other study inclusion/exclusion criteria apply

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You will be monitored continuously throughout the study for any changes in health or potential side effects.

Being in a clinical research study is voluntary and you can choose to leave at any time.
For more information about the ACCESS-1 Study:

Call +353 (0)61 533 400

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