Trial reportBlood2019
Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results.
Trial report in Blood, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 63 papers, 2 of them syntheses that pooled it.
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-Centre, Randomised, Open-Label, Controlled Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
A Multi-Centre Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Who cites it
63 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously treated individuals with haemophilia A or B.The Cochrane database of systematic reviews · 2021Pooled it
- A phase 1b/2 clinical study of marstacimab, targeting human tissue factor pathway inhibitor, in haemophilia.British journal of haematology · 2023Trial
- Long-term safety and efficacy of the anti-tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results.British journal of haematology · 2023Trial
- Non-Factor Therapies in Haemophilia: The Era of Factor VIII Mimetics and Targeted Rebalancing Agents.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Subcutaneous MG1113 in severe hemophilia A and B: phase 1b study for safety, pharmacokinetics, and pharmacodynamics.Research and practice in thrombosis and haemostasis · 2026Article
- Screening chemical libraries for the development of oral treatments for bleeding disorders.Blood vessels, thrombosis & hemostasis · 2026Article
- Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Review
- Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors.Journal of clinical medicine · 2025Review
- A Sponsor's Best Practice and Operating Principles to Manage Data Monitoring Committees.Therapeutic innovation & regulatory science · 2025Article
- Examining downstream effects of concizumab in hemophilia A with a mathematical modeling approach.Journal of thrombosis and haemostasis : JTH · 2025Article
- Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study.Research and practice in thrombosis and haemostasis · 2025Article
- Emerging Therapies in Hemophilia: Improving Equitable Access to Care.Journal of blood medicine · 2025Review
- Predicting a failure of postoperative thromboprophylaxis in non-small cell lung cancer: A stacking machine learning approach.PloS one · 2025Article
- Advances in Development of Drug Treatment for Hemophilia with Inhibitors.ACS pharmacology & translational science · 2024Review
- Thrombin generation to evaluate the complex hemostatic balance of hemophilia A plasma containing direct oral anticoagulant and supplemented by factor VIII.Research and practice in thrombosis and haemostasis · 2024Article
- Concizumab improves clot formation in hemophilia A under flow.Journal of thrombosis and haemostasis : JTH · 2024Article
- Current status and future perspectives of platelet-derived extracellular vesicles in cancer diagnosis and treatment.Biomarker research · 2024Review
- Current status and future prospects of activated recombinant coagulation factor VIIa, NovoSeven®, in the treatment of haemophilia and rare bleeding disorders.Annals of hematology · 2024Review
- Exploring the relationship between condition severity and health-related quality of life in people with haemophilia A across Europe: a multivariable analysis of data from the CHESS II study.Health and quality of life outcomes · 2024Article
3 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
15 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Results from the main parts (24 weeks) of 2 concizumab phase 2 trials are presented: explorer4 in hemophilia A (HA) or B (HB) with inhibitors (HAwI/HBwI) and explorer5 in HA. The trials aimed to evaluate the efficacy of daily subcutaneous concizumab prophylaxis (evaluated as annualized bleeding rate [ABR] at last dose level), with secondary objectives being safety and immunogenicity (assessed as number of adverse events [AEs] and antidrug antibodies [ADAs]). Patients received 0.15 mg/kg concizumab, with potential dose escalation to 0.20 and 0.25 mg/kg (if ≥3 spontaneous bleeding episodes within 12 weeks of concizumab treatment). Relevant pharmacokinetic/pharmacodynamic (PK/PD) parameters were assessed. Thirty-six HA, 9 HAwI, and 8 HBwI patients were exposed to concizumab. Most inhibitor patients (15 of 17; 88.2%) did not escalate the dose; all patients chose to continue to the extension phase of the trials. Clinical proof of concept for prevention of bleeding episodes was demonstrated in both trials. Estimated ABRs in HAwI and HBwI were lower vs HA: 3.0 (95% confidence interval [CI], 1.7; 5.3) and 5.9 (95% CI, 4.2; 8.5) vs 7.0 (95% CI, 4.6; 10.7), respectively. PK/PD results were as expected, with no difference between hemophilia subtypes for concizumab exposure, free tissue factor pathway inhibitor, thrombin generation, prothrombin fragment 1+2, and d-dimers. Concizumab was safe and well tolerated (no severe AEs, AE-related withdrawals, or thromboembolic events). Three patients had (very low to medium titer) ADA+ tests in each trial, with no observed clinical effect. These results support further development of concizumab as a daily prophylactic treatment in all hemophilia patients. These trials were registered at www.clinicaltrials.gov as #NCT03196284 and #NCT03196297.
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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.