NCT06411626Recruiting
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
Source: ClinicalTrials.gov · Last updated 27 Aug 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 128 (estimated)
- First submitted
- 8 May 2024
- Start date
- 11 Jun 2024
- Primary completion date
- 15 Jun 2026
- Lead sponsor
- Novartis Pharmaceuticals
- NCT number
- NCT06411626
What you commit to
- Study type: Observational
- Planned participants: 128
- From 11 Jun 2024 to the primary completion date, 15 Jun 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- 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.
Who the study is looking for
- 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
Age in the registry: 18 years to 99 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | East Hanover | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.