Danicopan Plus C5 Inhibitor Therapy in Paroxysmal Nocturnal Hemoglobinuria

Clinical Trial Title

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis

National Clinical Trial Number:


Contact Information

Rush Cancer Center Clinical Trials Office

Clinical Trial Protocol Description:

The name “paroxysmal nocturnal hemoglobinuria” (PNH) comes from: Paroxysmal – means “sudden and irregular;” Nocturnal – means “at night; Hemoglobinuria – means “hemoglobin in urine;” hemoglobin is the red part of red blood cells, it makes urine look dark. PNH is a rare and serious blood disease that causes red blood cells to break apart. Doctors call this breaking apart “hemolysis.” When red blood cells break apart, the hemoglobin inside is released. Hemoglobin is the red part of red blood cells that carries oxygen around the body.

People with PNH have blood cells that are missing a gene called PIG-A. This gene allows a substance called glycosyl-phosphatidylinositol (GPI) to help certain proteins stick to cells. Without PIG-A, important proteins cannot connect to the cell surface and protect the cell from substances in the blood called complement. As a result, red blood cells break down too early. Persistent hemolysis in people with PNH causes anemia, hemoglobinuria, and complications related to the presence of plasma free hemoglobin, including thrombosis, abdominal pain, dysphagia, erectile dysfunction, and pulmonary hypertension. Many patients with PNH are dependent on blood transfusions.

ALXN2040 (danicopan) is a complement system Factor D inhibitor. Factor D plays a key role in the activation of the alternative complement pathway that signals hemolysis in PNH. By blocking Factor D, ALXN2040 may help further treat PNH in patients currently receiving a C5 inhibitor.

This study will determine if ALXN2040 (danicopan), when used with a C5 inhibitor, improves anemia in patients with PNH.

Participants qualified for the study will be randomly assigned by chance (like the flip of a coin) to receive either study drug or placebo during Treatment period 1. They will have a 66% chance of receiving study drug and a 33% chance of receiving placebo. This is a double-blind study, which means the participant and the study doctor will both not know which of the study drug groups the participant is assigned to.

The study drug dosing interval is 3 times per day over the course of the 18-month study. During treatment periods 1 and 2, the participant will be dispensed 3 bottles and must take one pill from each of the bottles for each dose.

Clinical Trial Eligibility Criteria:

In order to participate you must meet the following criteria:

  • Are 18 years of age or older.
  • Have a diagnosis of PNH with Evident Extravascular hemolysis, which means anemia that requires at least 1 packed red blood cells (pRBCs) or whole blood transfusion within 6 months prior to the start of the study.
  • Are receiving an approved C5 inhibitor (eculizumab or ravulizumab) for at least 6 months prior to Day 1 in this study.

You will be excluded from the study if any of the following criteria apply to you:

  • Have a history of a major organ transplant (such as heart, lung, kidney, liver) or bone marrow transplant.
  • Have aplastic anemia or other bone marrow failure that requires a transplant or other therapies.
  • Have received another investigational agent other than C5 inhibitors within 30 days prior to study entry.
  • Have known underlying bleeding disorders.

This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.

Study Details

Clinical Trial Investigator

Jamile Shammo, MD

Contact Information

Rush Cancer Center Clinical Trials Office

Clinical Trial Location

Rush University Medical Center


Rush University Medical Center

1620 W Harrison St
Chicago, IL 60612

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