Capturing patient experiences in Paroxysmal Nocturnal Hemoglobinuria
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
This study is trying to gather information from people with Paroxysmal Nocturnal Hemoglobinuria about their symptoms and treatment experiences using a mobile app over six months to better understand their quality of life.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 128 (estimated) |
| Ages | 18 Years to 99 Years |
| Sex | All |
| Sponsor | Novartis Industry-sponsored |
| Locations | 1 site (East Hanover, New Jersey) |
| Trial ID | NCT06411626 on ClinicalTrials.gov |
What this trial studies
This observational study aims to longitudinally gather data on symptoms, treatment usage, and overall health-related quality of life (HRQoL) from patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). Participants will use the Folia mobile app to report their experiences, including symptom burden and treatment changes, over a six-month period. The study will employ a hybrid recruitment strategy, combining clinic and community referrals, and will include monthly survey check-ins to enhance data collection. The goal is to create a real-world data source that reflects the variability of symptoms and HRQoL in PNH patients, including those on specific therapies like iptacopan.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 or older with a diagnosis of PNH, regardless of their symptom severity or treatment history.
Not a fit: Patients who are not proficient in English or those outside the United States will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the real-world experiences of PNH patients, potentially leading to improved management strategies.
How similar studies have performed: While the approach of using home-reported outcomes is gaining traction, this specific study represents a novel effort to systematically capture patient experiences in PNH.
Eligibility criteria
Show full inclusion / exclusion 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 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.
Where this trial is running
East Hanover, New Jersey
- Novartis Investigative Site — East Hanover, New Jersey, United States (Recruiting)
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: +41613241111
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.