Hereditary Angioedema
87
3
12
62
Key Insights
Highlights
Success Rate
95% trial completion (above average)
Published Results
33 trials with published results (38%)
Research Maturity
62 completed trials (71% of total)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
3.4%
3 terminated out of 87 trials
95.4%
+8.9% vs benchmark
40%
35 trials in Phase 3/4
53%
33 of 62 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 62 completed trials
Clinical Trials (87)
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Berotralstat Treatment in Children With Hereditary Angioedema
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
Institutional Registry of Rare Diseases
A Study Observing US Patients With HAE Type I or II Who Take Icatibant to Treat HAE Attacks
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat