ALXN2040-PNH-301
Research type
Research Study
Full title
A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
IRAS ID
286382
Contact name
Austin Kulasekararaj
Contact email
Sponsor organisation
Alexion Pharmaceuticals Inc.
Eudract number
2019-003829-18
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
127,367, IND Number
Duration of Study in the UK
2 years, 11 months, 26 days
Research summary
This is a phase 3 Study in Patients with Paroxysmal Nocturnal Haemoglobinuria (PNH)who have clinically Evident Extravascular Haemolysis (EVH) on a C5 inhibitor (eculizumab or ravulizumab).
This study looks at an investigational drug called ALXN2040. This drug is being developed to treat PNH by blocking a protein called factor D in the complement system. By blocking factor D, ALXN2040 may help further treat PNH in participants currently receiving a C5 inhibitor but this is not yet proved. This study will determine if ALXN2040 (danicopan), when used with a C5 inhibitor, improves anaemia in participants with PNH.
Participants will be randomised to danicopan or placebo in a 2:1 ratio, for 12 weeks(Treatment Period 1) in addition to their C5 inhibitor therapy (eculizumab or ravulizumab).
At Week 12, participants randomised to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2) and participants randomised to danicopan will continue on danicopan for an additional 12 weeks, while remaining on the ongoing C5 inhibitor therapy.
At the end of the treatment periods (Week 24), participants may enter the Long-Term Extension (LTE) Period and continue to receive danicopan + their C5 inhibitor therapy.
About 100 study sites will be opened around the world to enroll approximately 84 participants.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
20/SC/0336
Date of REC Opinion
11 Sep 2020
REC opinion
Favourable Opinion