ALXN2040-PNH-303

• Research type

  Research Study

• Full title

  A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study.

• IRAS ID

  1005438

• Contact name

  Stephanie Haller

• Contact email

  stephanie.haller@alexion.com

• Sponsor organisation

  Alexion Pharmaceuticals Inc.

• Eudract number

  2021-004253-22

• Clinicaltrials.gov Identifier

  NCT05389449

• Research summary

  Participants that have previously participated in an Alexion parent study with danicopan for Paroxysmal Nocturnal Haemoglobinuria (PHN) and still have signs and symptoms of anaemia can participate in this study. PNH is a rare blood disease in which red blood cells (type of blood cells) are attacked by a part of the body’s immune system (system which defends your body against external attacks) known as the complement system. People with PNH produce red blood cells without key immune proteins (compound of your body) attached. Without these immune proteins, the complement system recognises the red blood cells as a threat and destroys them throughout the body. The destruction of red blood cells is largely responsible for many of the symptoms of PNH and causes a condition called anaemia.
  Currently, complement component 5 (C5) and complement component 3 (C3) inhibitors are the only approved treatment for PNH. C5 and C3 inhibitors work by suppressing the activity of a specific portion of the complement system. Some patients on approved C5 inhibitor therapies may continue to have anaemia. This study looks at an investigational drug called danicopan. This drug is being developed to treat PNH by blocking a protein called factor D in the complement system. By blocking factor D, danicopan may help further treat PNH in patients currently receiving a C5 inhibitor but this is not yet proved. The purpose of this study is to assess long-term efficacy and safety of danicopan in patients who continue to have anaemia while taking an approved C5 inhibitor. It is expected that approximately 89 study sites will be opened around the world and approximately 100 patients will participate in this study. The study will last up to 3 years and may include up to 15 visits.

• REC name

  South Central - Hampshire A Research Ethics Committee

• REC reference

  22/SC/0154

• Date of REC Opinion

  17 Jun 2022

• REC opinion

  Further Information Favourable Opinion