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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00456508 | Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE) | PHASE3 | COMPLETED | 147 | — | — | Apr 1, 2007 | Sep 1, 2010 | Jun 8, 2021 | 41 | United States, Canada +1 |
| NCT00457015 | Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE) | PHASE3 | COMPLETED | 96 | — | — | Apr 1, 2007 | Jun 1, 2008 | Jun 8, 2021 | 45 | United States, Canada +1 |
| NCT00262080 | Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE) | PHASE3 | COMPLETED | 91 | — | — | Dec 31, 2005 | Feb 28, 2007 | Jun 11, 2021 | 1 | United States |
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement \[100\] to significant worsening \[-100\]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.
| Arm | Type | Description |
|---|---|---|
| DX-88 (ecallantide) | EXPERIMENTAL | DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose. |
| Placebo | PLACEBO_COMPARATOR | Placebo, Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections. |
| Name | Type | Description |
|---|---|---|
| ecallantide | DRUG | solution for SC injection, one 30 mg dose per HAE attack |
| Phosphate Buffer Saline (PBS), pH 7.0 | DRUG | given as three 1mL subcutaneous injections. |
| Phosphate Buffer Saline (PBS), | DRUG | given as three 1mL subcutaneous injections. |
Inclusion Criteria: * 10 years of age or older * Documented diagnosis of HAE (Type I or II) * Willing and able to give informed consent * Acute HAE attack at time of presentation Exclusion Criteria: * Receipt of an investigational drug or device, within 30 days prior to study treatment, other tha...
| 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 |