A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Summary

Primary Objective: To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE Secondary Objective: To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE

Timeline

Start

2014-12

Primary completion

2016-05

Completion

2016-09

Outcome

Met primary endpoint

paper with mean differences of -4·4 attacks (p<0·0001) and -2·8 attacks (p=0·0004), respectively. PMID 28754491 ↗

Publications

• Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
• Riedl MA, Grivcheva-Panovska V, Moldovan D, Baker J, Yang WH, Giannetti BM, Reshef A, Andrejevic S, Lockey RF, Hakl R, Kivity S, Harper JR, Relan A, Cicardi M. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595-1602. doi: 10.1016/S0140-6736(17)31963-3. Epub 2017 Jul 25.

Drugs

Indications