Home Reported Outcomes in PNH
Recruiting
Observational Study
Paroxysmal Nocturnal Hemoglobinuria
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 18 – 99
- Sex
- Any
- Study type
- Observational
- Participants needed
- 128 (estimated)
- Sponsor
- Novartis Pharmaceuticals · Industry
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About This Trial
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 und…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
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 h…
Contacts
Novartis Pharmaceuticals
CONTACT