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NCT04469465 · ClinicalTrials.gov registry record · Phase 3
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
A Phase 3 study, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 86
- Enrollment target
NCT04469465: Completed Phase 3 study, sponsored by Alexion Pharmaceuticals.
NCT04469465 is a Phase 3 study that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 86 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (89% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04469465, a Phase 3 study, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 86 participants
- Enrollment target
Study 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.
Primary Outcome
Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.
Interventions
- DRUG Placebo
- DRUG Danicopan
- DRUG C5 Inhibitor
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 86 participants |
| Start Date | 2020-12-16 |
| Est. Completion | 2024-01-16 |
| Phase | Phase 3 |
What the finished NCT04469465 record still lists
NCT04469465 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 86 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (89% lower).
The record links to 0 conditions, and to 3 interventions - of which Placebo is the first listed.
NCT04469465 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04469465 about?
NCT04469465 is a clinical study titled "Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)". 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.
What is the current status of trial NCT04469465?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 86 participants. The study started on 2020-12-16. Estimated completion is 2024-01-16.
What interventions are being tested in trial NCT04469465?
The interventions under investigation include: Placebo (DRUG), Danicopan (DRUG), C5 Inhibitor (DRUG).
Who is sponsoring clinical trial NCT04469465?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04469465's enrollment target sits among peer trials
86 1745th of 2000 higher than 254 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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