Home Reported Outcomes in PNH
ConditionParoxysmal Nocturnal Hemoglobinuria
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-99
SponsorNovartis Pharmaceuticals
About this trial
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.
Eligibility criteria
Qualifiers
Aged 18 or older
US-based with a proficient understanding of and ability to read the English language
Any patient with a diagnosis of PNH, regardless of symptom or treatment history
Disqualifiers
None
Trial design
Treatments tested in this trial
- PNH-relevant therapies
Treatment groups
128 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov