Home › Paroxysmal Hemoglobinuria › 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

Who can join

Ages 18 to 99 · All sexes

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 history

Exclusion Criteria:

Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other exclusion criteria in order to meet the exploratory nature of the primary endpoint.

About the study

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.

What is being tested

• PNH-relevant therapies (other)

Sponsor: Novartis Pharmaceuticals · Participants: 128 · Started: Jun 11, 2024

Contact the study team

• Novartis Pharmaceuticals · Phone: +41613241111

Official record on ClinicalTrials.gov — NCT06411626

Locations in the U.S.

Conditions

• Paroxysmal Hemoglobinuria

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.