| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02052141 | Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema | PHASE3 | COMPLETED | 12 | — | — | Mar 20, 2014 | May 4, 2017 | Jun 3, 2021 | 8 | United States, Germany +3 |
Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.
| Arm | Type | Description |
|---|---|---|
| 500/1000 | EXPERIMENTAL | 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks |
| 1000/500 | EXPERIMENTAL | 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks |
| Name | Type | Description |
|---|---|---|
| CINRYZE 500 | BIOLOGICAL | 500 Units of CINRYZE administered by IV injection |
| CINRYZE 1000 | BIOLOGICAL | 1000 Units of CINRYZE administered by IV injection |
Inclusion Criteria: * Diagnosis of Type I or Type II HAE. * History of angioedema attacks. Exclusion Criteria: * History of bleeding or clotting abnormality. * Diagnosis of acquired angioedema or known to have C1 INH antibodies. * History of allergic reaction to C1 esterase inhibitor or other blo...
| 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 |