Trial reportBlood advances2022
Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study.
Trial report in Blood advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03896152 (A Multi-center, Randomized, Open-label, Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis), which is not on this map. Cited by 44 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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 Multi-center, Randomized, Open-label, Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis
Who cites it
44 citing papers in PubMed, 75 citations in OpenAlex.
- Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.Blood advances · 2026Trial
- Iptacopan for Immune Thrombocytopenia and Cold Agglutinin Disease: A Global Phase 2 Basket Clinical Trial.American journal of hematology · 2026Trial
- Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan.The patient · 2025Trial
- Assessment of QT Interval Prolongation Using Concentration-QT Modeling for Iptacopan, an Oral Complement Factor B Inhibitor, in Healthy Individuals.Clinical and translational science · 2025Trial
- Efficacy and safety of HRS-5965 monotherapy in complement inhibitor-naïve patients with paroxysmal nocturnal haemoglobinuria.British journal of haematology · 2025Trial
- Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of HSK39297 in Chinese Healthy Subjects: A Phase 1 Clinical Trial.Clinical and translational science · 2025Trial
- Article
- Molecular determinants of STEC-HUS: from complement activation to microvascular thrombosis.Frontiers in immunology · 2026Article
- New Anticomplement Drugs in Nephrology: Mechanism and Indication.Kidney & blood pressure research · 2026Review
- Efficacy analysis of iptacopan in a patient with thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: a case report.Frontiers in immunology · 2026Article
- Disease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations.Cost effectiveness and resource allocation : C/E · 2025Review
- Pharmacological Therapies in Paroxysmal Nocturnal Haemoglobinuria: Focus on Complement Inhibition.Drugs · 2025Review
- Advancements in complement inhibition for PNH and primary complement-mediated thrombotic microangiopathy.Blood advances · 2025Review
- Complement System Inhibitors in Nephrology: An Update-Narrative Review.International journal of molecular sciences · 2025Review
- The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria TreatmentTurkish journal of haematology : official journal of Turkish Society of Haematology · 2025Review
- Effect of Target-Mediated Disposition on Iptacopan Clinical Pharmacokinetics in Participants with Normal or Impaired Hepatic Function.Clinical pharmacology and therapeutics · 2025Article
- Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria.PharmacoEconomics · 2025Review
- Real-World Evidence of the Disease Burden and Economic Impact of Paroxysmal Nocturnal Hemoglobinuria in Italy.Journal of clinical medicine · 2025Article
- Clinical characteristics and management of paroxysmal nocturnal haemoglobinuria in Latin America: a narrative review.Annals of hematology · 2025Review
- Iptacopan Reduces Proteinuria and Stabilizes Kidney Function in C3 Glomerulopathy.Kidney international reports · 2025Article
Corrections and comments
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Authors and funding
10 authors at 8 institutions in 7 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH). In this ongoing open-label phase 2 study, PNH patients with active hemolysis were randomized to receive single-agent iptacopan twice daily at a dose of either 25 mg for 4 weeks followed by 100 mg for up to 2 years (cohort 1) or 50 mg for 4 weeks followed by 200 mg for up to 2 years (cohort 2). At the time of interim analysis, of 13 PNH patients enrolled, all 12 evaluable for efficacy achieved the primary endpoint of reduction in serum lactate dehydrogenase (LDH) levels by ≥60% by week 12 compared with baseline; mean LDH levels dropped rapidly and durably, namely by 77% and 85% at week 2 and by 86% and 86% at week 12 in cohorts 1 and 2, respectively. Most patients achieved a clinically meaningful improvement in hemoglobin (Hb) levels, and all but 1 patient remained transfusion-free up to week 12. Other markers of hemolysis, including bilirubin, reticulocytes, and haptoglobin, showed consistent improvements. No thromboembolic events were reported, and iptacopan was well tolerated, with no severe or serious adverse events reported until the data cutoff. In addition to the previously reported beneficial effect of iptacopan add-on therapy to eculizumab, this study showed that iptacopan monotherapy in treatment-naïve PNH patients resulted in normalization of hemolytic markers and rapid transfusion-free improvement of Hb levels in most patients. This trial was registered at www.clinicaltrials.gov as #NCT03896152.
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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.