drugset / Trial / NCT04307381
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study was to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with hereditary angioedema (HAE).
Timeline
- Start
- 2020-03-31
- Primary completion
- 2025-01-24
- Completion
- 2025-01-24
Publications
- Manning ME, de Lange M, Bernstein JA, Craig T, Lumry WR, Raasch J, Tachdjian R, Bordone L, Deng Y, Newman KB, Cohn DM. Donidalorsen for hereditary angioedema: Long-term results from a 4-year, phase 2, open-label extension study. Ann Allergy Asthma Immunol. 2026 Jun;136(6):699-705.e1. doi: 10.1016/j.anai.2026.02.019. Epub 2026 Mar 7.
- Petersen RS, Bordone L, Riedl MA, Tachdjian R, Craig TJ, Lumry WR, Manning ME, Bernstein JA, Raasch J, Zuraw BL, Deng Y, Newman KB, Alexander VJ, Lui C, Schneider E, Cohn DM. A phase 2 open-label extension study of prekallikrein inhibition with donidalorsen for hereditary angioedema. Allergy. 2024 Mar;79(3):724-734. doi: 10.1111/all.15948. Epub 2023 Nov 27.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Donidalorsen | Antisense oligonucleotide | 80 mg | Subcutaneous |
| Subject | Donidalorsen | Antisense oligonucleotide | 100 mg | Subcutaneous |