Deucrictibant for angioedema due to acquired C1-inhibitor deficiency : A randomized-controlled trial
Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved..
BACKGROUND: Angioedema due to acquired C1-inhibitor deficiency is a very rare but serious disease, with an estimated prevalence of 1 per 500,000 persons. There are no approved therapies to treat or prevent angioedema swelling in patients with this condition. Deucrictibant is a specific, orally bioavailable, competitive antagonist of the bradykinin B2 receptor currently under investigation for hereditary angioedema.
OBJECTIVE: Our aim was to assess the efficacy and safety of deucrictibant as acute and prophylactic treatment for angioedema due to acquired C1-inhibitor deficiency.
METHODS: A 2-part, randomized, double-blind, placebo-controlled crossover study was conducted. In Part 1, 4 consecutive angioedema attacks were treated with 3 doses of deucrictibant (10 mg, 20 mg, and 30 mg) or placebo. In Part 2, deucricibant, 20 mg, or placebo was administered twice daily for 2 treatment periods of 8 weeks.
RESULTS: Three patients were enrolled; of those 3 patients, 1 completed both study parts and 2 completed only Part 2. In Part 1, a reduction in attack severity was observed in the 3 attacks treated with deucrictibant as opposed to an increase in severity of the attack treated with placebo. In Part 2, the individual mean monthly attack rates were 2.0, 0.6, and 1.0 during the placebo period and 0.0 across all patients during treatment with deucrictibant. There were no severe adverse events and 1 self-limiting treatment-emergent adverse event (abdominal pain).
CONCLUSIONS: Deucrictibant has the potential to effectively and safely treat and prevent angioedema attacks due to acquired C1-inhibitor deficiency.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2024 |
|---|---|
| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - year:2024 |
|---|---|
| Enthalten in: |
The Journal of allergy and clinical immunology - (2024) vom: 15. März |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Petersen, Remy S [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Acquired C1-inhibitor deficiency |
|---|
| Anmerkungen: |
Date Revised 04.04.2024 published: Print-Electronic Citation Status Publisher |
|---|
| doi: |
10.1016/j.jaci.2024.03.007 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM369837827 |
|---|
| LEADER | 01000caa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM369837827 | ||
| 003 | DE-627 | ||
| 005 | 20240404235039.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 240318s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1016/j.jaci.2024.03.007 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1364.xml |
| 035 | |a (DE-627)NLM369837827 | ||
| 035 | |a (NLM)38494092 | ||
| 035 | |a (PII)S0091-6749(24)00271-9 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Petersen, Remy S |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Deucrictibant for angioedema due to acquired C1-inhibitor deficiency |b A randomized-controlled trial |
| 264 | 1 | |c 2024 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Revised 04.04.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status Publisher | ||
| 520 | |a Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a BACKGROUND: Angioedema due to acquired C1-inhibitor deficiency is a very rare but serious disease, with an estimated prevalence of 1 per 500,000 persons. There are no approved therapies to treat or prevent angioedema swelling in patients with this condition. Deucrictibant is a specific, orally bioavailable, competitive antagonist of the bradykinin B2 receptor currently under investigation for hereditary angioedema | ||
| 520 | |a OBJECTIVE: Our aim was to assess the efficacy and safety of deucrictibant as acute and prophylactic treatment for angioedema due to acquired C1-inhibitor deficiency | ||
| 520 | |a METHODS: A 2-part, randomized, double-blind, placebo-controlled crossover study was conducted. In Part 1, 4 consecutive angioedema attacks were treated with 3 doses of deucrictibant (10 mg, 20 mg, and 30 mg) or placebo. In Part 2, deucricibant, 20 mg, or placebo was administered twice daily for 2 treatment periods of 8 weeks | ||
| 520 | |a RESULTS: Three patients were enrolled; of those 3 patients, 1 completed both study parts and 2 completed only Part 2. In Part 1, a reduction in attack severity was observed in the 3 attacks treated with deucrictibant as opposed to an increase in severity of the attack treated with placebo. In Part 2, the individual mean monthly attack rates were 2.0, 0.6, and 1.0 during the placebo period and 0.0 across all patients during treatment with deucrictibant. There were no severe adverse events and 1 self-limiting treatment-emergent adverse event (abdominal pain) | ||
| 520 | |a CONCLUSIONS: Deucrictibant has the potential to effectively and safely treat and prevent angioedema attacks due to acquired C1-inhibitor deficiency | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Deucrictibant | |
| 650 | 4 | |a acquired C1-inhibitor deficiency | |
| 650 | 4 | |a acute treatment | |
| 650 | 4 | |a angioedema | |
| 650 | 4 | |a bradykinin B2 receptor | |
| 650 | 4 | |a long-term prophylaxis | |
| 650 | 4 | |a randomized controlled trial | |
| 700 | 1 | |a Fijen, Lauré M |e verfasserin |4 aut | |
| 700 | 1 | |a Kelder, Johannes P |e verfasserin |4 aut | |
| 700 | 1 | |a Cohn, Danny M |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t The Journal of allergy and clinical immunology |d 1971 |g (2024) vom: 15. März |w (DE-627)NLM000018449 |x 1097-6825 |7 nnns |
| 773 | 1 | 8 | |g year:2024 |g day:15 |g month:03 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1016/j.jaci.2024.03.007 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |j 2024 |b 15 |c 03 | ||