Hereditary Angioedema — Research Summary
Printed from RareWays (rareways.com.au) on 26 July 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Icatibant for acute hereditary angioedema attacks in pediatric patients: A systematized review.
Schmalzried Stephanie et al. — Allergy and asthma proceedings (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42343494/
- 2.
Hereditary angioedema attack trends among patients maintained on lanadelumab long-term prophylaxis.
O'Connor Maeve et al. — The journal of allergy and clinical immunology. Global (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42328483/
- 3.
Sebetralstat for on-demand treatment of hereditary angioedema: A pooled analysis of placebo-controlled clinical trials.
Aygören-Pürsün Emel et al. — The World Allergy Organization journal (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42318595/
- 4.
Long-term prophylaxis in hereditary angioedema: Real-world treatment patterns and healthcare resource utilization.
Tachdjian Raffi et al. — The World Allergy Organization journal (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42318594/
- 5.
Characterization of hereditary angioedema population in Argentina: A nationwide study.
Josviak Darío et al. — The World Allergy Organization journal (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42292763/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
Recruiting — Phase 3 — Pharvaris Netherlands B.V.
https://clinicaltrials.gov/study/NCT06679881
- 2.
Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)
Recruiting — Phase 3 — ADARx Pharmaceuticals, Inc.
https://clinicaltrials.gov/study/NCT07428499
- 3.
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
Recruiting — Phase 3 — ADARx Pharmaceuticals, Inc.
https://clinicaltrials.gov/study/NCT06960213
- 4.
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant IR Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
Recruiting — Phase 3 — Pharvaris Netherlands B.V.
https://clinicaltrials.gov/study/NCT07266805
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Hereditary Angioedema
Hereditary angioedema is a rare genetic condition causing recurrent episodes of severe swelling in the skin, abdomen, and airway. It is caused by deficiency or dysfunction of C1 inhibitor. Untreated attacks can be life-threatening. Effective preventive and on-demand treatments are now available on the PBS.
Most Recent Research
Background: Hereditary angioedema (HAE) is a rare, potentially life-threatening bradykinin-mediated disorder with limited pediatric treatment options and little comparative evidence. Icatibant, a bradykinin B2 receptor antagonist, has demonstrated efficacy in adults and has emerging evidence in children. Objective: To evaluate and synthesize clinical evidence on the efficacy and safety of icatibant for acute HAE attacks in pediatric patients. Methods: A literature search was performed by using medical literature data bases, clinical trial and research registries, and key journals through January 2026. The primary outcome was the time to onset of symptom relief. Secondary outcomes included the time to minimum symptoms, pain scores, adverse events, and feasibility of self- or caregiver administration. All included studies were nonrandomized, single-arm trials with heterogeneous designs and outcomes definitions; therefore, a meta-analysis was not performed, and all studies were descriptively evaluated. Results: This systematic review identified three clinical studies of icatibant for acute HAE attacks in children ages 0 to 17 years. Three open-label, phase III studies, which included 43 pediatric patients were reviewed. Icatibant demonstrated rapid symptom relief, with a median onset at ∼1 hour and minimum symptoms within 2 hours, although repeated dosing showed variability. Icatibant was well tolerated, with injection-site reactions as the most common adverse events and no serious treatment-related events. Self- or caregiver administration was feasible and effective in adolescents. Conclusion: Icatibant was safe and effective in the pediatric studies, providing rapid symptoms relief and a favorable tolerability profile. Despite limitations from small sample sizes and lack of comparator trials, current evidence supports icatibant as a targeted, practical option for treating acute HAE attacks in children.
Common Questions
What is Hereditary Angioedema?
Hereditary angioedema is a rare genetic condition causing recurrent episodes of severe swelling in the skin, abdomen, and airway. It is caused by deficiency or dysfunction of C1 inhibitor. Untreated attacks can be life-threatening. Effective preventive and on-demand treatments are now available on the PBS.
How many clinical trials are available for Hereditary Angioedema?
RareWays currently indexes 155 clinical trials for Hereditary Angioedema, of which 14 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Hereditary Angioedema come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.
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This information is for general awareness only.
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