Home Reported Outcomes in PNH
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: PNH-relevant therapies.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria. Basic parameters: 18 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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.
Detailed description
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.
Interventions
- Other PNH-relevant therapies
This is an observational study. There is no treatment allocation. The decision to initiate PNH-relevant therapies (such as eculizumab, ravulizumab, pegcetacoplan, iptacopan, and others) will be based solely on clinical judgement.
Primary outcome measures
- PNH symptom burden [Time frame: Baseline, month 6]
Secondary outcome measures (3)
- Number of participants by treatment utilization and management of potential flare events [Time frame: Up to 6 months]
- Number of participants by reported treatment use and treatment switching [Time frame: Up to 6 months]
- Health-related quality of life (HRQoL) [Time frame: up to 6 months]
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 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Novartis Investigative Site — East Hanover
Identifiers
NCT: NCT06411626 · CLNP023C1US01