CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 86 enrolled
Drug / intervention
Danicopan +2 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04469465
NCT04469465Phase 3Completed

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)

Alexion Pharmaceuticals, Inc.·interventional·Posted Jul 14, 2020·Updated May 4, 2025

In Brief

A Phase 3 clinical trial evaluating Danicopan, Placebo, and 1 other intervention for Paroxysmal Nocturnal Hemoglobinuria. Completed, enrolled 86 participants across 62 sites in 18 countries.

Detailed Summary

The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesBrazil, Canada, Czechia, France, Germany, Greece, Israel, Italy, Japan, Malaysia, Netherlands, Poland, South Korea, Spain, Taiwan, Thailand, United Kingdom, United States
Collaborators--

Timeline

Phase 3CompletedFinished
202120222023202420252026
First PostedJul 14, 2020
Enrollment StartDec 16, 2020
Primary CompletionJun 29, 2022
Study CompletionJan 16, 2024
TodayJul 2, 2026
Enrollment to primary: 1.5 yearsPosted 6.0 years ago

Interventions

Danicopandrug

Oral tablet

Placebodrug

Oral tablet

C5 Inhibitordrug

Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.