Non-therapeutic Study If you are interested in learning more about your possible participation in this study, please complete the form. Your information will be forwarded directly to the study sponsor.
The IRB-approved observational Phase IV PLEDGE study is set-up to collect real-world data on adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) with symptomatic extravascular hemolysis who recently started VOYDEYATM (danicopan) as add-on treatment to ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab).
The Study will collect & analyze patient-reported outcome (PRO) assessments related to fatigue and quality of life and short custom surveys using a digital patient platform and electronic medical records from adult patients with PNH and extravascular hemolysis. Study duration will be about six months from initiation of danicopan treatment.
Patients enrolled in the study will be compensated for their time to complete digital patient-reported outcome (PRO) measurements and short custom surveys.
- Paroxysmal Nocturnal Hemoglobinuria (PNH) diagnosis confirmed in the medical record
- Initiation of VOYDEYA™ (danicopan) subsequent to signing the informed consent or, in patients who already initiated danicopan within 7 days
- The index use of danicopan is in combination with ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab)
- Age 18 years or older
- Ability to give informed consent in English
- History of VOYDEYA™ (danicopan) treatment that was initiated at least six months prior to their initial PNH diagnosis
- History of danicopan treatment in the 14 days prior to informed consent
- Current use of pegcetacoplan, crovalimab, or iptacopan
- Active bone marrow failure at the time of enrollment