Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
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|ClinicalTrials.gov Identifier: NCT01188564|
Recruitment Status : Completed
First Posted : August 25, 2010
Results First Posted : August 7, 2015
Last Update Posted : August 7, 2015
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Angioedema||Drug: rhC1INH Drug: Placebo (Saline)||Phase 3|
HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.
rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.
Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.
Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||75 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase III Randomized, Double-blind, Placebo-controlled Study With an Open-label Extension Evaluating the Efficacy, Safety and Immunogenicity of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks of Angioedema in Patients With HAE|
|Study Start Date :||January 2011|
|Actual Primary Completion Date :||December 2012|
|Actual Study Completion Date :||March 2013|
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 Comparator: Placebo (Saline)||
Drug: Placebo (Saline)
One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment
- Time to Beginning of Relief of Symptoms [ Time Frame: Patients observed for 24 hours ]
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 Minimal Symptoms [ Time Frame: 24 hours ]The key secondary efficacy endpoint was the time to minimal symptoms at all locations. The time to achieving minimal symptoms was defined as an answer of "Yes" to TEQ question 3.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01188564