Trial reportThe patient2025
Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan.
Trial report in The patient, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody.
An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan
A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
Who cites it
1 citing paper in PubMed.
- Recommendations for the use of clinical outcome assessments in rare disease drug development.EClinicalMedicine · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundParoxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disease, with symptoms including fatigue, difficulty breathing, and hemoglobinuria, which negatively affect health-related quality of life.
objectiveIn-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.
methodsAdult 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.
resultsInterviews 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.
conclusionFindings 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).
Indexed as
Identifiers
40694294What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.