Trial reportOrphanet journal of rare diseases2025
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study.
Trial report in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04364269 (A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects With Non-transfusion Dependent β-thalassaemia), which is not on this map. Cited by 3 papers.
What it found
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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 Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects With Non-transfusion Dependent β-thalassaemia
Who cites it
3 citing papers in PubMed.
- Hepcidin as a Molecular Hub of Iron Homeostasis: From BMP-SMAD Signaling to Therapeutic Modulation.Biomolecules · 2026Review
- Identification and characterization of novel hepcidin mimetics that modulate ferroportin function.Blood red cells & iron · 2026Article
- Molecular and therapeutic frontiers in anemia therapy.The Journal of clinical investigation · 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
9 authors.
Funding
Abstract
backgroundNon-transfusion-dependent beta-thalassemia (β-NTDT) is characterized by ineffective erythropoiesis, increased intestinal iron absorption, and iron overload. The ferroportin inhibitor, vamifeport, has been shown to improve erythropoiesis via decreases in serum iron and transferrin saturation levels in preclinical models and healthy volunteer studies.
objectiveThe objective of this 12-week, double-blind, randomized, placebo-controlled, phase 2a study was to assess the safety and tolerability of vamifeport versus placebo in adults with β-NTDT (primary endpoint). Iron-related pharmacodynamic effects (preliminary efficacy) were also assessed as a secondary endpoint.
methodsRandomized, adult patients weighing 40-59 kg and 60-100 kg received vamifeport 60 mg and 120 mg (once [QD] or twice [BID] daily), respectively, for 12 weeks. Non-transfusion-dependent thalassemia was defined as transfusion requirements < 5 units of red blood cells during the 24 weeks before randomization.
resultsTwenty-five patients were included (vamifeport QD n = 9, BID n = 12; placebo n = 4); 64% were male and 56% weighed < 60 kg. Baseline serum iron and transferrin saturation levels were similar across treatment groups. All treatment-emergent adverse events were mild/moderate, and rates were similar across groups (vamifeport QD 67%, BID 58%; placebo 75%). There were no deaths or serious treatment-emergent adverse events and no clinically relevant changes in safety parameters. Serum iron and transferrin saturation levels decreased by 2 h after the first vamifeport dose (mean [standard deviation] decreased QD - 12.2 [6.5], BID - 14.5 [12.1] µmol/L and QD - 33.6 [18.9], BID - 37.2 [27.6] %, respectively) and remained below baseline levels at each subsequent visit. There were no clinically meaningful changes in the placebo group.
conclusionIn this 12-week study, vamifeport had a favorable safety/tolerability profile, with no changes in hemoglobin levels ≥ 1.0 g/dL, and promising pharmacodynamic effects versus placebo in adults with β-NTDT.
trial registrationClinicalTrials.gov, NCT04364269. Registered 01 April 2020; Prospectively registered, https://clinicaltrials.gov/study/NCT04364269?term=NCT04364269&rank=1 .
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