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. 2025 Jul 22.
doi: 10.1007/s40271-025-00755-5. Online ahead of print.

Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan

Carlos DeCastro  1 , Phillip Sheinberg  2 , Bing Han  3 , Susan Vallow  4 , Georgina Bermann  5 , Marion Dhalke  6 , Rakesh Kumar  6 , Gavin Dickie  7 , Nina Galipeau  7 , Roger Lamoureux  7 , Kaelyn Rupinski  7 , Caitlyn Lowe  7 , Amber Nieves  7 , Flore Sicre de Fontbrune  8 , Regis Peffault de Latour  9
Affiliations

Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan

Carlos DeCastro et al. Patient. .

Abstract

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disease, with symptoms including fatigue, difficulty breathing, and hemoglobinuria, which negatively affect health-related quality of life.

Objective: In-trial interviews with patients with PNH, enrolled across three clinical trials, were conducted to understand patient experiences and satisfaction after receiving iptacopan, a novel treatment for PNH.

Methods: Adult participants with PNH were recruited and consented into the qualitative interviews across three phase II/III iptacopan trials (NCT04558918, NCT04820530, and NCT04747613). Interview documents were developed and approved by the relevant ethics authorities in target countries. Trained qualitative interviewers used a semi-structured interview guide to elicit information on PNH-related symptoms and impacts participants experienced prior to the trial, how these changed after receiving iptacopan, and their treatment experience and satisfaction with iptacopan.

Results: Interviews were conducted with 61 participants in eight countries. Participants reported a broad range of symptoms and impacts associated with PNH, including fatigue, tiredness, frustration, difficulty doing usual activities, and limitation to social activities. Interview participants reported improvements in the signs, symptoms, and impacts of PNH after receiving iptacopan, and the majority of participants considered those improvements to be meaningful. Participants reported being either "very satisfied" or "satisfied" with their treatment experience of iptacopan.

Conclusion: Findings from these interviews provide valuable patient-reported data on the positive treatment experience and meaningful improvements in PNH symptoms and health-related quality-of-life impacts patients reported after receiving iptacopan. These qualitative reports from patients support and contextualize the positive efficacy results demonstrated in the three iptacopan clinical trials. NCT04558918 (16 Sep 2020), NCT04820530 (25 Mar 2021), NCT04747613 (09 Feb 2021).

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Conflict of interest statement

Declarations. Funding: The research reported here, and the development of this manuscript, were supported by funding from Novartis. Conflict of Interest: Gavin Dickie, Nina Galipeau, Roger Lamoureux, Kaelyn Rupinski, and Caitlyn Lowe are employees of Adelphi Values, which was compensated by Novartis AG for the conduct of the research reported here. Amber Nieves was an employee of Adelphi Values at the time the research reported here was conducted. Ethics Approval: Ethics approval was received for all patient interviews. Country and site-specific ethics approval was obtained for the conduct of patient experience interviews as part of the relevant clinical trial protocol approvals. Author Contributions: All authors contributed to the study conception and design. Gavin Dickie, Nina Galipeau, Roger Lamoureux, Kaelyn Rupinski, Caitlyn Lowe, and Amber Nieves prepared material preparation and collected data. Gavin Dickie, Nina Galipeau, Roger Lamoureux, Kaelyn Rupinski, Caitlyn Lowe, Amber Nieves, Susan Vallow, Georgina Bermann, Marion Dhalke, and Rakesh Kumar analyzed the data. Roger Lamoureux wrote the first draft of the manuscript, and all authors commented on subsequent versions. All authors read and approved the final manuscript. Data Availability: The data that support the findings of this study are not publicly available due to reasons of sensitivity but are available from the corresponding author upon reasonable request.

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