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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00262301 | Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema | PHASE3 | COMPLETED | 75 | — | — | Jun 1, 2004 | Oct 1, 2009 | Oct 2, 2012 | 2 | Netherlands, Romania |
| NCT02247739 | A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor | PHASE2 | COMPLETED | 32 | — | — | Dec 1, 2014 | Sep 1, 2016 | Dec 8, 2017 | 10 | United States, Canada +5 |
| NCT00225147 | Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema | PHASE2 | COMPLETED | 77 | — | — | Jul 1, 2005 | Jan 1, 2010 | Feb 22, 2013 | 1 | Netherlands |
The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
Average number of HAE attacks normalized to a 28 day period
| Arm | Type | Description |
|---|---|---|
| 100 IU/kg "rhC1INH" | EXPERIMENTAL | 100 IU/kg recombinant human C1 inhibitor |
| Saline | PLACEBO_COMPARATOR | Saline solution |
| rhC1INH twice weekly | EXPERIMENTAL | rhC1INH administered twice weekly |
| rhC1INH once weekly | EXPERIMENTAL | rhC1INH administered once weekly |
| Placebo (Saline) twice weekly | PLACEBO_COMPARATOR | Placebo (Saline) administered twice weekly |
| 100 IU/kg rhC1INH | EXPERIMENTAL | 100 IU/kg Recombinant human C1 inhibitor |
| 50 IU/kg rhC1INH | EXPERIMENTAL | 50 IU/kg Recombinant human C1 inhibitor |
| Name | Type | Description |
|---|---|---|
| recombinant human C1 inhibitor | DRUG | IV |
| Placebo | DRUG | IV |
Inclusion Criteria: * Clear clinical and laboratory diagnosis of HAE * Baseline plasma level of functional C1INH of less than 50% of normal * Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack Exclusion Cri...
| 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 |