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Also known as C1 esterase inhibitor [human] liquid
C1 esterase inhibitor liquid · 1 trial · 1 indication
The angioedema attacks were recorded in the electronic patient diary. The investigator completed a separate angioedema eCRF for each attack based on the review of the patients diary. Time-normalized number of angioedema attacks was expressed as the number of attacks per month (ie, 30.4 days) of exposure. NNA=30.4 \* (number of attacks during treatment period) / (days of treatment period).
| Arm | Type | Description |
|---|---|---|
| Experimental/Placebo | EXPERIMENTAL | Subjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period. |
| Placebo/Experimental | EXPERIMENTAL | Subjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period. |
| Experimental/ Experimental | EXPERIMENTAL | Subjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period |
| Name | Type | Description |
|---|---|---|
| C1 esterase inhibitor [human] liquid | DRUG | C1 Esterase Inhibitor \[Human\] Liquid administered Subcutaneously as specified on specified days |
| Placebo | DRUG | Placebo |
The maximum duration of participation is approximately 9 months. Patients will complete a screening period of up to 21 days. Following screening, eligible patients will be randomly assigned to 1 of 3 treatment sequences. Each patient will undergo 2 14-week treatment periods for a total of 28 weeks (...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002 |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | Deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
C1 esterase inhibitor liquid is being developed for the prevention of angioedema attacks in adolescents and adults with hereditary angioedema, or HAE. HAE is an immunology condition characterized by recurrent swelling attacks. The investigational therapy is administered subcutaneously as a preventive treatment in this patient population.
C1 esterase inhibitor liquid is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor of the clinical program evaluating this subcutaneous therapy for hereditary angioedema. The company is responsible for the development and regulatory strategy for the asset.
C1 esterase inhibitor liquid is in Phase 3 clinical development for hereditary angioedema. The Phase 3 study evaluating the therapy has been completed. The drug remains investigational and has not been approved by the FDA for any indication based on the available development information.
C1 esterase inhibitor liquid has been evaluated in one Phase 3 clinical trial, NCT02584959, titled a study to evaluate the clinical efficacy and safety of subcutaneously administered C1 esterase inhibitor for the prevention of angioedema attacks in adolescents and adults with hereditary angioedema. The trial enrolled 75 participants across the United States, Canada, Germany, Hungary, Israel, Romania, and Spain, and it has been completed.
Yes, C1 esterase inhibitor liquid is also known as C1 esterase inhibitor [human] liquid. Both names refer to the same investigational therapy being developed by Takeda for hereditary angioedema. The bracketed human designation describes the human-derived source of the C1 esterase inhibitor protein used in the liquid formulation.
The Phase 3 trial NCT02584959 was a randomized, double-blind, controlled study evaluating subcutaneous C1 esterase inhibitor for preventing angioedema attacks in adolescents and adults with hereditary angioedema. It enrolled 75 participants aged 12 years and older, across both sexes, and was conducted in seven countries. The study has been completed.