Clinical Trial: Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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 Ac
Brief Summary: This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.
Detailed Summary:
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.
Sponsor: Pharming Technologies B.V.
Current Primary Outcome: 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.
Original Primary Outcome: Time to beginning of relief based on the VAS, completed by the patient [ Time Frame: Patients observed for at least 8 hours ]
Current Secondary Outcome: Time to Minimal Symptoms [ Time Frame: 24 hours ]
Original Secondary Outcome:
- Minimal important difference (MID) of the overall VAS score
- Time to beginning of relief based on the MID
- Time to beginning of relief based on the Investigator Score
- IgM and IgG antibodies against recombinant human C1INH (anti-rhC1INH)
- Antibodies against host related impurities (anti-HRI)
- Adverse events
Information By: Pharming Technologies B.V.
Dates:
Date Received: August 24, 2010
Date Started: January 2011
Date Completion:
Last Updated: August 3, 2015
Last Verified: July 2015