Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
Paroxysmal nocturnal hemoglobinuria, PNH, home reported outcomes, HRO, patient-reported outcome, PRO, real-world data, iptacopan
Brief summary
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.
Detailed description
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.
Interventions
OTHERPNH-relevant therapies
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.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
Study participants eligible for inclusion in this study must meet all of the following criteria: * 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
Exclusion criteria
Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| PNH symptom burden | Baseline, month 6 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants by treatment utilization and management of potential flare events | Up to 6 months | Treatment utilization and management of potential flare events tracked during the study period |
| Number of participants by reported treatment use and treatment switching | Up to 6 months | HRO-reported treatment use and treatment switching throughout the study duration, triangulated with symptoms reported throughout the study duration |
| Health-related quality of life (HRQoL) | up to 6 months | HRQoL HRO tracking data: * Mood & behavioral health * Productivity * Reasons for discontinuing or changing a treatment |
Countries
United States
Contacts
Primary ContactNovartis Pharmaceuticals
Backup ContactNovartis Pharmaceuticals
Outcome results
None listed