Trial reportClinical and translational science2025
Exposure-Response Analysis of Donidalorsen for the Treatment of Hereditary Angioedema.
Trial report in Clinical and translational science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05139810 (A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema), which is not on this map. Cited by 3 papers.
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 Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)
Who cites it
3 citing papers in PubMed.
- Exposure-Response Analysis of Donidalorsen for the Treatment of Hereditary Angioedema.Clinical and translational science · 2025Trial
- Population Pharmacokinetic/Pharmacodynamic Modeling of Donidalorsen, an Antisense Oligonucleotide in Development for Prophylaxis of Hereditary Angioedema.CPT: pharmacometrics & systems pharmacology · 2026Article
- Donidalorsen for the Treatment of Hereditary Angioedema: A Review of Clinical Studies.Drug design, development and therapy · 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
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hereditary angioedema (HAE) is characterized by recurrent attacks of severe tissue swelling. In the OASIS-HAE phase 3 study (NCT05139810), donidalorsen, an RNA-targeted antisense oligonucleotide that reduces prekallikrein production in the liver, significantly reduced HAE attack rates. To characterize the relationship between prekallikrein concentrations and HAE attack rates following donidalorsen and predict the efficacy of potential dosing regimens, an exposure-response model was developed using data from OASIS-HAE. Simulations were conducted to evaluate the following regimens: 80 mg once every 4 weeks (Q4W), 8 weeks (Q8W), 1 month (Q1M), and 2 months (Q2M), and a switch to Q2M dosing for patients who were attack-free for 3 months on the Q1M regimen. The relationship between prekallikrein concentrations and HAE attack rates was well characterized by a sigmoidal E
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