Hereditary Angioedema
88
2
9
65
Key Insights
Highlights
Success Rate
96% trial completion (above average)
Published Results
37 trials with published results (42%)
Research Maturity
65 completed trials (74% of total)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
3.4%
3 terminated out of 88 trials
95.6%
+9.0% vs benchmark
41%
36 trials in Phase 3/4
57%
37 of 65 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 65 completed trials
Clinical Trials (88)
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)
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
PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial
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)
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Berotralstat Treatment in Children With Hereditary Angioedema
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
Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema