drugset / Trial / NCT04618211

Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

Phase 2 Completed 74 enrolled Pharvaris Netherlands B.V.

RandomizedCrossoverQuadruple-blindTreatment

Summary

This study evaluates the efficacy of orally administered deucrictibant for the acute treatment of attacks in patients with hereditary angioedema (HAE). Eligible subjects are randomized to one of three single doses of deucrictibant and placebo. The study will compare symptom relief (skin pain, skin swelling, abdominal pain) during HAE attacks and safety of each dose of deucrictibant with placebo.

Timeline

Start: 2021-02-03
Primary completion: 2022-09-23
Completion: 2023-03-01

Outcome

Met primary endpoint

registry PHA-022121 Low Dose - 10 mg vs Placebo; p < 0.0001; Least squares mean difference -16.75 (95% CI -21.52 to -11.97) NCT04618211 ↗

registry PHA-022121 Medium Dose - 20 mg vs Placebo; p < 0.0001; Least squares mean difference -15.02 (95% CI -20.22 to -9.81) NCT04618211 ↗

registry PHA-022121 High Dose - 30 mg vs Placebo; p < 0.0001; Least squares mean difference -16.28 (95% CI -21.27 to -11.29) NCT04618211 ↗

paper Deucrictibant significantly reduced the severity of hereditary angioedema attacks compared with placebo PMID 41865747 ↗

Publications

Maurer M, Stobiecki M, Valerieva A, Hakl R, Staevska MT, Bouillet L, Du-Thanh A, Kessel A, Kiani-Alikhan S, Magerl M, Reshef A, Baeza ML, Fain O, Farkas H, Greve J, Guilarte M, Jacobs JS, Li HH, Lleonart R, Manning ME, Sussman GL, Anderson J, Chapdelaine H, Cohn DM, Hagin D, Kralickova P, Ritchie B, Spadaro G, Staubach P, Tarzi MD, Yang WH, Jouvin MH, Crabbe R, van Leeuwen S, Chen H, Zhu L, Knolle J, Lesage A, Lu P, Riedl MA, Aygoren-Pursun E. Oral deucrictibant for on-demand treatment of hereditary angioedema attacks (RAPIDe-1): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2026 Apr;13(4):e200-e214. doi: 10.1016/S2352-3026(25)00341-2. Epub 2026 Mar 19.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CRICTIBANT	Small molecule	10 mg	Oral
Subject	CRICTIBANT	Small molecule	20 mg	Oral
Subject	CRICTIBANT	Small molecule	30 mg	Oral