NCT06411626

Home Reported Outcomes in PNH

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 128 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
OTHER	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.

Timeline

• Start date: 2024-06-11
• Primary completion: 2026-06-15
• Completion: 2026-06-15
• First posted: 2024-05-13
• Last updated: 2025-08-27

Locations

1 site across 1 country: United States