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Home › Conditions › Hereditary Angioedema

Hereditary Angioedema

Part of Hypersensitivity, Immediate

6 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Hereditary Angioedema

35 trials tracked for Hereditary Angioedema: 24 in phase 3 or 4 and 8 with published results. The most-cited published study has 164 citations.

35Trials tracked
24Phase 3 & 4
0Recruiting
8With published results
Phase distribution
Phase 4 2 Phase 3 22 Phase 2 11
  1. Phase 3 Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE Completed · 164 cited
  2. Phase 3 CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks Completed · 116 cited
  3. Phase 3 OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) Completed · 68 cited
  4. Phase 3 A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE) Completed · 63 cited
  5. Phase 3 A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children Completed · 29 cited
  6. Phase 3 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks Completed · 24 cited
Show 29 more trials
  1. Phase 3 A Long Term Safety Study of BCX7353 in Hereditary Angioedema Completed · 13 cited
  2. Phase 3 Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks Completed · 9 cited
  3. Phase 4 Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE Completed
  4. Phase 4 A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE Completed
  5. Phase 3 A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema Completed
  6. Phase 3 Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema Completed
  7. Phase 3 Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks Completed
  8. Phase 3 A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3) Completed
  9. Phase 3 Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks Completed
  10. Phase 3 Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks Completed
  11. Phase 3 C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks Completed
  12. Phase 3 C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study) Completed
  13. Phase 3 EASSI - Evaluation of the Safety of Self-Administration With Icatibant Completed
  14. Phase 3 Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema (HAE) Completed
  15. Phase 3 C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks Completed
  16. Phase 3 Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema Completed
  17. Phase 3 Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks Completed
  18. Phase 3 Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema Completed
  19. Phase 2 An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema Completed
  20. Phase 2 A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration Completed
  21. Phase 2 A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE) Completed
  22. Phase 2 A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration Completed
  23. Phase 2 Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients Completed
  24. Phase 2 A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II Completed
  25. Phase 2 Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Completed
  26. Phase 2 A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema Completed
  27. Phase 2 A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase Completed
  28. Phase 2 A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema Completed
  29. Phase 2 A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor Completed

Showing the 35 most-cited and recently-updated of 35 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Hereditary Angioedema: what the trials found

Several therapies have demonstrated significant efficacy in reducing the frequency and severity of Hereditary Angioedema (HAE) attacks. CSL312 showed a 95.41% reduction in attack rates during the treatment period compared to the run-in period 1, with a statistically significant reduction in time-normalized HAE attacks per month compared to placebo 7. Donidalorsen also demonstrated a significant reduction in investigator-confirmed HAE attack rates (p<0.001) and increased the proportion of patients who were attack-free from week 5 to week 25 8.

Acute treatment options have shown rapid relief for HAE symptoms. Icatibant significantly reduced time to symptom relief, primary symptom relief, and almost complete symptom relief compared to placebo in multiple trials [16, 18], with some studies reporting very low VAS scores for symptom intensity 4. Recombinant human C1 inhibitor (rhC1INH) also demonstrated significant improvements in the time to beginning of relief of symptoms and time to minimal symptoms across several studies [19, 20, 22].

Other interventions include BCX7353, which showed a statistically significant reduction in the rate of investigator-confirmed HAE attacks over a 24-week period 6, and BCX4161, which was evaluated for its impact on attack-free days and disease activity scores 11.

Placebo data indicates that patients without active intervention experienced significantly longer times to symptom relief and resolution compared to those receiving active treatment in specific trial designs 9.

Recent results — preliminary, needs further review

  • Navenibart showed a substantial reduction in monthly HAE attack rates (ranging from -84% to -88%) and high proportions of attack-free days in a Phase 2 study 3.
  • Navenibart was also associated with significant reductions in plasma levels of cleaved High-molecular-weight Kininogen (cHMWK) 3.

For the clinician treating this condition

  • CSL312 and Donidalorsen are established as effective options for reducing the frequency of HAE attacks over a treatment period.
  • Icatibant and recombinant human C1 inhibitor provide rapid, statistically significant relief from acute HAE symptoms.
  • Navenibart shows promising early data in Phase 2 trials regarding both attack rate reduction and biochemical markers (cHMWK).

AI synthesis of 8 cited trials, updated Jun 28, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Regulatory and financing barriers primarily hinder optimal care delivery for hereditary angioedema in Balkan countries
Allergy & Immunology Guideline
Regulatory and financing barriers primarily hinder optimal care delivery for hereditary angioedema in Balkan countries Policy gaps create hurdles for patients with hereditary angioedema
This policy and practice review analyzes health systems and care delivery for hereditary angioedema across the Balkan Peninsula. The analysi…
Patients with hereditary angioedema face significant barriers to care, including long wait times for diagnosis and limited access to modern …
Frontiers Jul 21, 2026
New HAE subtypes involving endothelial dysfunction support biomarker discovery and therapeutic strategies to stabilize the vascular barrier
Allergy & Immunology Sys. Review
New HAE subtypes involving endothelial dysfunction support biomarker discovery and therapeutic strategies to stabilize the vascular barrier New insights into hereditary angioedema mechanisms and treatment strategies
This review discusses implications for biomarker discovery and therapeutic strategies aimed at stabilizing the endothelial barrier in patien…
A review discusses how stabilizing the endothelial barrier could help treat hereditary angioedema, offering new therapeutic directions for p…
Frontiers Jun 1, 2026
Probiotic co-administration with berotralstat improves GI tolerability in HAE patients
Gastroenterology Cohort
Probiotic co-administration with berotralstat improves GI tolerability in HAE patients Probiotics may help manage side effects of HAE treatment
This pilot case series from six Italian centers evaluated probiotic co-administration with berotralstat in 25 adolescents and adults with he…
New early treatment steps for hereditary angioedema might reduce monthly attacks while keeping stomach issues to a minimum.
Frontiers Apr 30, 2026
Lanadelumab reduced attacks in Chinese adults with hereditary angioedema over six months
Allergy & Immunology Phase III
Lanadelumab reduced attacks in Chinese adults with hereditary angioedema over six months Imagine waking up with severe swelling in your throat. It feels like you cannot breathe. This is what hereditary angioedema feels like for many people
This open-label study evaluated safety and efficacy of subcutaneous lanadelumab in patients with hereditary angioedema in China.
This rare condition causes painful swelling under the skin.
Frontiers Apr 29, 2026
Oral deucrictibant reduced hereditary angioedema attack rates versus placebo in a phase 2 trial
Allergy & Immunology RCT
Oral deucrictibant reduced hereditary angioedema attack rates versus placebo in a phase 2 trial Can a daily pill prevent painful swelling attacks for people with hereditary angioedema?
A phase 2 randomized trial in 34 adults with hereditary angioedema types 1 or 2 found that daily oral deucrictibant (20 mg or 40 mg) signifi…
A daily pill reduced painful hereditary angioedema attacks by 80% in adults taking it for 12 weeks, offering a new preventive option for thi…
Apr 5, 2026
Oral deucrictibant reduces hereditary angioedema attack severity in phase 2 crossover trial
Allergy & Immunology RCT
Oral deucrictibant reduces hereditary angioedema attack severity in phase 2 crossover trial Oral drug reduces hereditary angioedema attack severity in phase 2 trial
In a phase 2 randomized, double-blind, placebo-controlled crossover trial of 62 adults with hereditary angioedema, oral deucrictibant (10 mg…
A new oral drug reduced the severity of hereditary angioedema attacks compared to placebo, offering a convenient treatment option for painfu…
Apr 5, 2026

Research across Hypersensitivity, Immediate

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Peri- and postmenopausal hormonal changes may exacerbate asthma, rhinitis, cough, allergies, hypersensitivity, anaphylaxis, and angioedema
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Peri- and postmenopausal hormonal changes may exacerbate asthma, rhinitis, cough, allergies, hypersensitivity, anaphylaxis, and angioedema Menopause Changes Allergy Rules for Women
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Questions about Hereditary Angioedema

Did lanadelumab reduce the number of attacks for adults with Hereditary Angioedema in China?

Yes, lanadelumab reduced monthly HAE attacks by 99.1% in a Chinese phase 3 study, with 90% of patients attack-free over six months.

Full answer →