Home Reported Outcomes in PNH

NCT06411626 · tracked via the Priya Life Science USA tracker

Sponsor

Novartis Pharmaceuticals

Phase

Observational

Started

2024-06-11

Last updated

2025-08-27

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Investigational drug(s) / intervention(s)

PNH-relevant therapies

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.

Study summary

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

Sex

ALL

Min age

18 Years

Max age

99 Years

Healthy volunteers

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.

Primary outcome measure(s)

PNH symptom burden — Baseline, month 6
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.

Trial sites (1)

Facility	City	Region	Status
Novartis Investigative Site	East Hanover	New Jersey	Recruiting

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Other trials for the same condition

Long-term Safety of Danicopan: IPIG Registry-based Cohort Study Active, not recruiting

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors Active, not recruiting

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria Active, not recruiting

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment Active, not recruiting

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06411626 on ClinicalTrials.gov ↗ ← All trials in the USA