Actively Recruiting
Home Reported Outcomes in PNH
Led by Novartis Pharmaceuticals · Updated on 2025-08-27
128
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Home Reported Outcomes in PNH
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 18 or older
- US-based and able to read and understand English
- Diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH), regardless of symptoms or treatment history
You will not qualify if you...
- Not meeting all the inclusion criteria above
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Novartis Investigative Site
East Hanover, New Jersey, United States, 07936
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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