Non-therapeutic Study Non-therapeutic Study: The purpose of a non-therapeutic study is to increase the understanding of a disease or condition through observation, data collection, or other methods. A non-therapeutic study does not provide treatment or intervention for participants. This type of research contributes to general knowledge that may benefit others in the future by improving treatment or developing new therapies. 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 Paroxysmal Nocturnal Hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) with symptomatic extravascular hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. 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 danicopan: VOYDEYA is a prescription medicine used along with ravulizumab or eculizumab to treat breakdown of red blood cells that takes place outside of blood vessels (extravascular hemolysis), in adults with paroxysmal nocturnal hemoglobinuria (PNH). What is VOYDEYA? VOYDEYA is a… 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 Paroxysmal Nocturnal Hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) diagnosis confirmed in the medical record
- Initiation of VOYDEYA™ (danicopan) subsequent to signing the informed consent or, in patients who already initiated danicopan danicopan: VOYDEYA is a prescription medicine used along with ravulizumab or eculizumab to treat breakdown of red blood cells that takes place outside of blood vessels (extravascular hemolysis), in adults with paroxysmal nocturnal hemoglobinuria (PNH). What is VOYDEYA? VOYDEYA is a… 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 danicopan: VOYDEYA is a prescription medicine used along with ravulizumab or eculizumab to treat breakdown of red blood cells that takes place outside of blood vessels (extravascular hemolysis), in adults with paroxysmal nocturnal hemoglobinuria (PNH). What is VOYDEYA? VOYDEYA is a… treatment in the 14 days prior to informed consent
- Current use of pegcetacoplan pegcetacoplan: EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given skin (subcutaneously) by using the Empaveli injector or with an… , crovalimab crovalimab: An experimental complement inhibitor C5 monoclonal antibody. By blocking the cleavage of C5 to C5a and C5b, it is expected to inhibit complement activation, which is the cause of a number of diseases. , or iptacopan iptacopan: FABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It was approved by the U.S. Food and Drug Administration in December 2023. Fabhalta is taken twice a day in a capsule form. What is FABHALTA? …
- Active bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… at the time of enrollment