Actively Recruiting
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
Led by Novartis Pharmaceuticals · Updated on 2025-08-27
128
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying Paroxysmal Nocturnal Hemoglobinuria (PNH) to understand the range of symptoms, treatment use, and overall health-related quality of life experienced by patients with this condition. The study focuses on collecting real-world data, especially from home-reported outcomes, to capture symptom changes and treatment patterns, including for those using orally administered iptacopan. Participants will use the Folia mobile app to enroll, consent, and report their symptoms, treatments, and quality of life over six months. The study is observational, meaning treatments such as eculizumab, ravulizumab, pegcetacoplan, or iptacopan will be used based on clinical decisions and not assigned by the study. Monthly surveys and possible integration of health records will supplement data collection. During the study, participants will regularly track their symptoms and treatment changes through the app. Researchers will measure PNH symptom burden at the start and after six months, as well as monitor treatment use, management of flare events, treatment switching, and quality of life. The study aims to provide a comprehensive view of living with PNH over this half-year period.
CONDITIONS
Brief Title
Home Reported Outcomes in PNH
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 18 or older
- US-based with a proficient understanding of and ability to read the English language
- Any patient with a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), regardless of symptom or treatment history
You will not qualify if you...
- Does not meet all the inclusion criteria above
- No other exclusion criteria due to the exploratory nature of the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit (virtual via mobile app)
Duration - 6 months
Participants use a mobile app to track routine treatment, symptoms, changes in treatment plans, and health-related quality of life over time.
Monthly survey check-ins via mobile app
Trial Site Locations
Total: 1 location
1
Novartis Investigative Site
East Hanover, New Jersey, United States, 07936
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
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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