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
Location: See location...
Intervention Type: Other
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 99
Healthy Volunteers: f
View:

⁃ 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

Locations
United States
New Jersey
Novartis Investigative Site
RECRUITING
East Hanover
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
+41613241111
Backup
Novartis Pharmaceuticals
Time Frame
Start Date: 2024-06-11
Estimated Completion Date: 2026-06-15
Participants
Target number of participants: 128
Treatments
Participants with PNH
Participants with PNH
Sponsors
Leads: Novartis Pharmaceuticals

This content was sourced from clinicaltrials.gov