Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as rhC1INH
i.v. recombinant human C1 inhibitor · 4 trials · 2 indications
Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which * The patient reports any of the following answers for TEQ question 1: "A little better", "Better" or "Much better"; and; * The patient reports the following answer for TEQ question 2: "Yes"; and, * There is persistence in improvement at the next assessment time, i.e.either the same or a better response to Question 1 and "Yes" to Question 2.
Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).
| Arm | Type | Description |
|---|---|---|
| rhC1INH | EXPERIMENTAL | - |
| Placebo (Saline) | PLACEBO_COMPARATOR | - |
| Recombinant Human C1 Inhibitor | EXPERIMENTAL | Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U. |
| Recombinant Human C1INH | EXPERIMENTAL | - |
| 1 | OTHER | Open-label i.v. administration of 100 U/kg rhC1INH |
| Name | Type | Description |
|---|---|---|
| rhC1INH | DRUG | One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater. |
| Placebo (Saline) | DRUG | One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment |
| i.v. recombinant human C1 inhibitor | DRUG | - |
Inclusion Criteria: * Aged at least 13 years * Signed written informed consent * Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal * Willingness and ability to comply with all protocol procedures * Clinical symptoms of an eligib...
Top 7 of 8 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 4 | PHASE3 | Fazirsiran |
| Rhythm Pharmaceuticals, Inc. | RYTM | 1 | PHASE1 | RM-718 |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE1 | ALN-SOD |
| Tango Therapeutics, Inc. | TNGX | 1 | PHASE1 | TNG462 |
| Cogent Biosciences, Inc. | COGT | 1 | PHASE1 | CGT4859 |
| Kura Oncology, Inc. | KURA | 2 | PHASE1 | Ziftomenib |
| Wave Life Sciences Ltd. | WVE | 1 | PHASE1 | WVE-006 |
I.v. recombinant human C1 inhibitor is an investigational therapy being studied for the treatment of acute attacks in patients with hereditary angioedema, a genetic disorder. It is administered intravenously and is currently in Phase 2 clinical development.
I.v. recombinant human C1 inhibitor is being developed by Pharming Group N.V., a biopharmaceutical company listed on the stock exchange under the ticker symbol PHAR.
I.v. recombinant human C1 inhibitor is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Two Phase 2 trials have been completed, but the drug remains in clinical development.
I.v. recombinant human C1 inhibitor has been studied in two completed Phase 2 clinical trials: NCT00261053 and NCT00262288. Both trials evaluated the drug for the treatment of acute attacks in patients with hereditary angioedema and were conducted in the Netherlands.
I.v. recombinant human C1 inhibitor is a recombinant form of the human C1 inhibitor protein. It is designed to replace the deficient or dysfunctional C1 inhibitor in patients with hereditary angioedema, thereby helping to control acute attacks of the condition.