OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

Phase 3 Completed 91 enrolled Ionis Pharmaceuticals, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).

Timeline

Start: 2021-12-03
Primary completion: 2023-11-09
Completion: 2023-11-09

Outcome

Met primary endpoint

registry analysis (superiority test); Pooled Placebo vs Cohort A: Donidalorsen 80 mg; p <0.001; IC HAE attack rate ratio 0.19 NCT05139810 ↗

registry analysis (superiority test); Pooled Placebo vs Cohort B: Donidalorsen 80 mg; p =0.004; IC HAE attack rate ratio 0.45 NCT05139810 ↗

Publications

Results Riedl MA, Tachdjian R, Lumry WR, Craig T, Karakaya G, Gelincik A, Stobiecki M, Jacobs JS, Gokmen NM, Reshef A, Gompels MM, Manning ME, Bordone L, Newman KB, Treadwell S, Wang S, Yarlas A, Cohn DM; OASIS-HAE Team. Efficacy and Safety of Donidalorsen for Hereditary Angioedema. N Engl J Med. 2024 Jul 4;391(1):21-31. doi: 10.1056/NEJMoa2402478. Epub 2024 May 31.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Donidalorsen	Antisense oligonucleotide	80 mg	Subcutaneous