ClinicalTrials.gov record 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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This prospective observational study asks adults with PNH to use the Folia mobile app to report symptoms, treatments, and health-related quality of life. Each participant is followed for six months with monthly surveys; the study may also link reports with electronic health record or claims data. No treatments are assigned by the study—clinical care decisions remain with participants' clinicians.
What it is studying. The study is creating a real-world dataset of home-reported outcomes in PNH to describe symptom burden and variability over time, patterns of treatment use and switching (including orally administered iptacopan), and health-related quality of life and management of potential flares.
What the researchers hope to learn. To establish a real-world data source describing longitudinal symptom burden, treatment utilization and switching, management of potential flares, and health-related quality of life in people with PNH.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Other
- PNH-relevant therapies
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
The study will be prospective and observational, conducted over an initial period of six months per individual from the point of study enrollment. Participants will utilize the Folia mobile app to enroll, consent, and complete all study activities. A hybrid recruitment method of clinic referrals and community referrals will be employed to identify participants, who will be asked to track routine treatment, symptoms, changes in treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins will be conducted to capture additional data inputs, with the possibility of integrating electronic health record (EHR) and/or claims data.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Use the Folia mobile app to enroll, give consent, and complete study activities. Regularly report routine treatments, symptoms, any treatment changes, and health-related quality of life through the app, with monthly survey check-ins over an initial six-month period. The study may also link participants' electronic health record (EHR) and/or claims data if available. This is an observational study; there is no study-assigned treatment.
How the study is structured. A prospective, observational cohort run through the Folia mobile app. Participants enroll and consent in the app, complete monthly surveys and patient-reported outcome measures, and track routine treatments, symptom changes, and HRQoL over a six-month period. The study does not provide or assign treatments; treatment decisions are made by participants and their clinicians. The study may also integrate participants' electronic health record or claims data if available.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- Jun 11, 2024
- Primary completion
- Jun 15, 2026 (estimated)
- Study completion
- Jun 15, 2026 (estimated)
- Target enrollment
- 128 participants (estimated)
- Last updated on registry
- Aug 27, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Novartis Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novartis Pharmaceuticals 📞 +41613241111 ✉ novartis.email@novartis.com
- Novartis Pharmaceuticals
Participating sites (1)
- Novartis Investigative SiteRecruiting
East Hanover, New Jersey, 07936 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- primary endpoint
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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