Home Reported Outcomes in PNH

Part of paid clinical trials in East Hanover, New Jersey.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT06411626
Status
Recruiting
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Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • PNH-relevant therapies — OTHER
    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.

Study Details

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.

Key Dates

Start date
Jun 11, 2024
Status verified
Aug 2025
Primary completion
Jun 15, 2026
Completion
Jun 15, 2026

Study Design

Enrollment
128 participants (estimated)

Arms

  • Arm: Participants with PNH
    Participants with PNH

Primary Outcome Measure

PNH symptom burden [ Time Frame: Baseline, month 6 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Novartis Investigative SiteEast HanoverNew Jersey07936-

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Novartis Investigative Site· East Hanover, NJ

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