*ALXN2040-PNH-302 Open label study of Danicopan as add-on treatment in adolescents with PNH
Research type
Research Study
Full title
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
IRAS ID
1010297
Contact name
Gleb Filippov
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc
Clinicaltrials.gov Identifier
Research summary
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare blood disease in which red blood cells are attacked by a part of the body’s immune system known as the complement system. People with PNH produce red blood cells without key immune proteins 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, C5 inhibitors are the only approved treatment for PNH in paediatric patients. C5 inhibitors work by suppressing the activity of a specific portion of the complement system. The two C5 inhibitors available are eculizumab (Soliris®) and ravulizumab (Ultomiris®). Some patients on approved C5 inhibitor therapies may continue to have anaemia.
This study looks at an investigational drug called ALXN2040 (Danicopan). This drug is being developed to treat PNH in paediatric patients by blocking a protein called factor D in the complement system. By blocking factor D, Danicopan may help further treat PNH in paediatric patients currently receiving a C5 inhibitor, but this is not yet proved in paediatric patients. This study will determine if Danicopan when used with a C5 inhibitor, improves anaemia in paediatric patients with PNH.
The purpose of this study is to assess the efficacy and safety of Danicopan in paediatric patients who continue to have anaemia on an approved C5 inhibitor. About 10 study sites will be opened around the world to enrol about 6 patients.
This study will consist of 6 parts and will last up to 64 weeks.
REC name
London - Brent Research Ethics Committee
REC reference
24/LO/0702
Date of REC Opinion
9 Dec 2024
REC opinion
Further Information Favourable Opinion