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.
The study will be prospective and observational, conducted over an initial period of six months per individual from the point of study enrollment. Participants will utilize the Folia mobile app to enroll, consent, and complete all study activities. A hybrid recruitment method of clinic referrals and community referrals will be employed to identify participants, who will be asked to track routine treatment, symptoms, changes in treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins will be conducted to capture additional data inputs, with the possibility of integrating electronic health record (EHR) and/or claims data.
Study Type
OBSERVATIONAL
Enrollment
128
This is an observational study. There is no treatment allocation. The decision to initiate PNH-relevant therapies (such as eculizumab, ravulizumab, pegcetacoplan, iptacopan, and others) will be based solely on clinical judgement.
Novartis Investigative Site
East Hanover, New Jersey, United States
RECRUITINGPNH symptom burden
Primary endpoint will be measured through longitudinal Home Reported Outcomes (HRO) tracking of self-selected symptoms at enrollment and associated changes in severity against self-reported baselines.
Time frame: Baseline, month 6
Number of participants by treatment utilization and management of potential flare events
Treatment utilization and management of potential flare events tracked during the study period
Time frame: Up to 6 months
Number of participants by reported treatment use and treatment switching
HRO-reported treatment use and treatment switching throughout the study duration, triangulated with symptoms reported throughout the study duration
Time frame: Up to 6 months
Health-related quality of life (HRQoL)
HRQoL HRO tracking data: * Mood \& behavioral health * Productivity * Reasons for discontinuing or changing a treatment
Time frame: up to 6 months
Novartis Pharmaceuticals
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