It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
Hereditary angioedema is a rare, potentially life-threatening genetically determined disease associated with a deficiency or impairment of the functional activity of the C1-esterase inhibitor (C1-inhibitor). The main clinical manifestation of hereditary angioedema is recurrent subcutaneous or submucosal swelling of various localization. Most often, the development of the disease is based on a mutation in the SERPING1 gene. The prevalence of the disease in the world ranges from 1:10 000 to 1:150 000. GNR-038 is a recombinant C1 inhibitor (rhC1INH), which is a complete structural and functional analog of the plasma C1 inhibitor. Phase I study results showed convincing safety and tolerability evidence of GNR-038.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
A single intravenous infusion of GNR-038, 50 МЕ/ kg less than 5 hours after the onset of edema.
A single intravenous infusion of GNR-038, 100 МЕ/ kg less than 5 hours after the onset of edema.
A single intravenous infusion of Berinert®, 20 МЕ/ kg less than 5 hours after the onset of edema.
National Research Center - Institute of Immunology Federal Medical-Biological Agency of Russia
Moscow, Russia
Moscow City Clinical Hospital 52
Moscow, Russia
Federal State Budgetary Scientific Institution Research Institute of Fundamental and Clinical Immunology
Moscow, Russia
Rostov State Medical University
Rostov-on-Don, Russia
Time to symptoms relief onset of acute HAE attack within 24 hours after the end of the drug administration.
A persistent decrease in the intensity of symptoms by 20 mm from the initial level on the visual analogue scale (VAS) will be regarded as a relief of symptoms of HAE. 0 mm is the absence of symptoms, 100 mm is the maximum possible intensity of symptoms
Time frame: 24 hours
Time to complete resolution of the symptoms of an acute HAE attack within 24 hours after the end of the study drug administration.
Persistent absence of symptoms - 0 (zero) mm on the visual analogue scale (VAS). 0 mm is the absence of symptoms, 100 mm is the maximum possible intensity of symptoms
Time frame: 24 hours
Time to minimum manifestation onset of acute HAE attack symptoms after the completion of study drug administration.
Persistent reduction in the intensity of symptoms below 20 (twenty) mm on the visual analogue scale(VAS). 0 mm is the absence of symptoms, 100 mm is the maximum possible intensity of symptoms
Time frame: 24 hours
The proportion of HAE exacerbation episodes that achieved symptom relief after 1 hour and 4 (four) hours after the end of study drug administration.
Time frame: 1 hour; 4 hours.
The rate of attacks with HAE current localization relapse or with the occurrence of a new acute attack of a different localization within 24 (twenty-four) hours after the study drug administration.
Time frame: 24 hours
The rate of attacks that required additional administration of emergency drugs (human C1-esterase inhibitor or icatibant).
Time frame: 24 hours
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A single intravenous infusion of Placebo less than 5 hours after the onset of edema.
A single intravenous infusion of GNR-038 less than 5 hours after the onset of edema.
A single intravenous infusion of Berinert®, 20 МЕ/ kg less than 5 hours after the onset of edema.
LLC "Scientific Medical Center of General Therapy and Pharmacology"
Stavropol, Russia
The rate of attacks that did not respond therapeutically to the study drug administration
the lack of relief of HAE symptoms within 4 (four) hours after administration of the drug, the occurrence of a relapse of HAE of the current localization or the occurrence of a new acute attack of another localization within 24 (twenty-four) hours after drug administration, the need to use drugs that can weaken the symptoms of HAE (see drugs that are not recommended to treatment), within 24 (twenty-four) hours after drug administration.
Time frame: 4 hours; 24 hours
The intensity of acute HAE attack symptoms within 24 (twenty-four) hours after study drug administration.
HAE intensity wiil be measured by visual analogue scale (VAS). 0 mm is the absence of symptoms, 100 mm is the maximum possible intensity of symptoms
Time frame: 24 hours
Frequency of adverse events.
Time frame: 14 days
Frequency of anti-drug antibody formation.
Time frame: 7 and 14 days
The level of anti-drug antibodies neutralizing activity.
Laboratory measurement of antidrug antibody with neutralixing activity.
Time frame: 7 and 14 days