Home Reported Outcomes in PNH
ConditionParoxysmal Nocturnal Hemoglobinuria
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-99
SponsorNovartis Pharmaceuticals
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
None
Trial population
Individuals aged 18+ who have been diagnosed with PNH, regardless of symptom or treatment history, and are US-based with a proficient understanding of and ability to read the English language.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
PNH-relevant therapies
Other interventionThis 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.
Treatment groups
128 Participants
are divided into 1 treatment groupGroup A: Participants with PNH1 intervention
Trial outcomes
Primary outcomes
1
PNH symptom burden
Primary endpoint will be measured through longitudinal Home Reported Outcomes (HRO) tracking of self-selected symptoms at enrollment and associated changes in severity against self-reported baselines.
Time frame
Baseline, month 6Secondary outcomes
1
Number of participants by treatment utilization and management of potential flare events
Treatment utilization and management of potential flare events tracked during the study period
Time frame
Up to 6 months2
Number of participants by reported treatment use and treatment switching
HRO-reported treatment use and treatment switching throughout the study duration, triangulated with symptoms reported throughout the study duration
Time frame
Up to 6 months3
Health-related quality of life (HRQoL)
HRQoL HRO tracking data: * Mood \& behavioral health * Productivity * Reasons for discontinuing or changing a treatment
Time frame
up to 6 monthsSponsors and contacts
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Source ClinicalTrials.gov