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NCT07413250 · ClinicalTrials.gov registry record
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
A clinical trial of Paroxysmal Nocturnal Hemoglobinuria and PNH, sponsored by Alexion Pharmaceuticals.
- Active
- Registry status
- 50
- Enrollment target
- 1
- Study location
NCT07413250: Active study of Paroxysmal Nocturnal Hemoglobinuria and PNH, sponsored by Alexion Pharmaceuticals.
NCT07413250 is a study of Paroxysmal Nocturnal Hemoglobinuria and PNH that is active but no longer recruiting, run by Alexion Pharmaceuticals. The registered enrollment target is 50 participants, below the 261-participant average among 14 other Paroxysmal Nocturnal Hemoglobinuria trials with a reported enrollment target (81% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07413250, a study of Paroxysmal Nocturnal Hemoglobinuria and PNH, is active but no longer recruiting, sponsored by Alexion Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- 50 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.
Conditions Studied
Interventions
- DRUG Danicopan
- DRUG Soliris/Ultomiris
Study Locations (1)
Massachusetts
- Clinical Research Site - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2026-01-14 |
| Est. Completion | 2030-01-15 |
What the registry record for NCT07413250 still lists
NCT07413250 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target, below the 261-participant average among 14 other Paroxysmal Nocturnal Hemoglobinuria trials with a reported enrollment target (81% lower).
The record links to 2 conditions, with Paroxysmal Nocturnal Hemoglobinuria appearing as the primary indexed condition, and to 2 interventions - of which Danicopan is the first listed.
NCT07413250 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT07413250 about?
NCT07413250 is a clinical study titled "Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data". This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of ...
What is the current status of trial NCT07413250?
This trial is currently active not recruiting. The enrollment target is 50 participants. The study started on 2026-01-14. Estimated completion is 2030-01-15.
What conditions does trial NCT07413250 study?
This clinical trial studies the following conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH.
What interventions are being tested in trial NCT07413250?
The interventions under investigation include: Danicopan (DRUG), Soliris/Ultomiris (DRUG).
Who is sponsoring clinical trial NCT07413250?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07413250 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Paroxysmal Nocturnal Hemoglobinuria
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT07413250's enrollment target sits among peer trials
50 10th of 14 higher than 3 of 14 other Paroxysmal Nocturnal Hemoglobinuria trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Paroxysmal Nocturnal Hemoglobinuria 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
-
The International PNH Interest Group PNH Registry
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Home Reported Outcomes in PNH
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A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
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Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
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