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Home Reported Outcomes in PNH
Sponsor: Novartis Pharmaceuticals
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.
Official title: Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US
Key Details
Gender
All
Age Range
18 Years - 99 Years
Study Type
OBSERVATIONAL
Enrollment
128
Start Date
2024-06-11
Completion Date
2026-06-15
Last Updated
2025-08-27
Healthy Volunteers
No
Conditions
Interventions
PNH-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.
Locations (1)
Novartis Investigative Site
East Hanover, New Jersey, United States