Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06467084 | Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II | PHASE3 | COMPLETED | 36 | — | — | Jun 24, 2024 | Jan 15, 2026 | Jan 23, 2026 | 24 | United States, Canada +5 |
| NCT05505916 | 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) | PHASE3 | COMPLETED | 145 | — | — | Oct 24, 2022 | May 27, 2026 | Jun 5, 2026 | 71 | United States, Australia +21 |
| NCT05511922 | PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial | PHASE3 | COMPLETED | 11 | — | — | Oct 24, 2022 | May 27, 2026 | Jun 8, 2026 | 61 | United States, Australia +15 |
| NCT05259917 | 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) | PHASE3 | COMPLETED | 136 | — | — | Feb 22, 2022 | Dec 31, 2023 | May 2, 2025 | 66 | United States, Australia +19 |
| NCT04208412 | 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 | PHASE2 | COMPLETED | 84 | — | — | Jul 2, 2019 | Dec 8, 2020 | May 2, 2025 | 25 | United States, Austria +8 |
| NCT04349800 | A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers | PHASE1 | COMPLETED | 84 | — | — | Jan 4, 2018 | Sep 10, 2018 | Apr 29, 2025 | 1 | United Kingdom |
The analysis of time to the beginning of symptom relief defined as at least "a little better" (2 time points in a row) on the PGI-C within 12 hours of the first IMP administration using the Gehan score transformation test for Full Analysis Set (FAS). Attacks were treated as right-censored at 12 hours if they did not achieve beginning of symptom relief defined by PGI-C as at least "a little better" (2 time points in a row) or received conventional attack treatment prior to time-to-event within 12 hours of the first IMP administration. When an endpoint result was non-evaluable (NE) within 12 hours, if the event did occur, the event must have occurred \>12 hours following study drug.
The primary variable for statistical comparison between treatments in Part 2 of the study was time to use of conventional attack treatment (pdC1INH or rhC1INH intravenous \[iv\] or icatibant) within 12 hours of study drug. Censoring occurs where a subject did not use conventional attack treatment within 12h post-study drug dosing. When an endpoint result was non-calculable (NC) within 12 hours, if the event did occur, the event must have occurred \>12 hours following study drug.
| Arm | Type | Description |
|---|---|---|
| 150 mg Dose Group | OTHER | Patients will take a single 150 mg dose of KVD900. |
| 300 mg Dose Group | OTHER | Patients will take a single 300 mg dose of KVD900. |
| 600 mg Dose Group | OTHER | Patients will take a single 600 mg dose of KVD900. |
| KVD900 600 mg | EXPERIMENTAL | - |
| KVD900 300 mg | EXPERIMENTAL | - |
| Experimental: KVD900 300 mg | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Part 1 | EXPERIMENTAL | Subjects received a single dose of 600 mg KVD900. |
| Part 2 - Sequence 1: 600 mg KVD900, Then Placebo | EXPERIMENTAL | Subjects received a single dose of 600 mg KVD900 to treat the first eligible HAE attack. Following resolution of this attack, subjects received a second single dose of placebo to treat the second eligible HAE attack. |
| Part 2 - Sequence 2: Placebo, Then 600 mg KVD900 | EXPERIMENTAL | Subjects received a single dose of placebo to treat the first eligible HAE attack. Following resolution of this attack, subjects received a second single dose of 600 mg KVD900 to treat the second eligible HAE attack. |
| Single Ascending Dose - 5 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 10 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 20 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 40 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 80 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 160 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 300 mg | EXPERIMENTAL | - |
| Single Ascending Dose - 600 mg | EXPERIMENTAL | - |
| Formulation Screen | EXPERIMENTAL | - |
| Food Effect | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| KVD900 150 mg | DRUG | KVD900 Tablet 150 mg (2 x 75 mg) |
| KVD900 300 mg | DRUG | KVD900 Tablet 300 mg (1 x 300 mg) |
| KVD900 600 mg | DRUG | KVD900 Tablet 600 mg (2 x 300 mg) |
| Drug: KVD900 300 mg | DRUG | KVD900 Tablet 300 mg |
| Placebo | DRUG | Placebo to KVD900 Tablet |
| KVD900 | DRUG | KVD900 tablet 600 mg |
| Placebo to KVD900 | DRUG | Placebo |
Inclusion Criteria: 1. Male or female patients 2 to 11 years of age. 2. Confirmed diagnosis of HAE Type I or II. 3. For patients ≥20 kg at screening, patient has had at least 1 documented HAE attack in the last year prior to screening. 4. Caregiver, as assessed by the Investigator, must be able to ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration, Biological NTLA-2002 |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat, berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| KalVista Pharmaceuticals, Inc. | KALV | 1 | PHASE3 | KVD900, Drug: KVD900 |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |