Prevention of angioedema attacks in children 6 to 11 years of age with hereditary angioedema. MedDRA version: 16.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: CINRYZE®
Pharmaceutical Form: Powder and solvent for solution for injection
Current Sponsor code: VP 20624
Other descriptive name: COMPLEMENT C1 ESTERASE INHIBITOR
Concentration unit: U
Sponsors
ViroPharma Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Be a child (male of female), =6 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Have a history of hypercoagulability (abnormal blood clotting). 2. Have a diagnosis of acquired angioedema or known to have C1 INH antibodies. 3. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products. 4. Be pregnant or breastfeeding. 5. Have received an investigational drug other than dose required for prevention or treatment of angioedema attacks within 30 days prior to screening. 6. Have, as determined by the Investigator and the Sponsor's medical monitor, any surgical or medical condition that could interfere with the administration of study drug or interpretation of study results. Additional Exclusion Criteria (Disqualifying from Randomization) 7. Have any active infectious illness or fever defined as an oral temperature >38 oC (100.4 oF), tympanic >38.5 oC (101.3 oF), axillary >38 oC (100.4 oF), or rectal/core >38.5 oC(101.3 oF) within 24 hours prior to the first dose of study drug in Treatment Period 1. 8. Have had signs or symptoms of an angioedema attack within 2 days prior to the first dose of study drug in Treatment Period 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the relative efficacy of two dose levels of CINRYZE (500 U and 1000 U) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age.;Secondary Objective: SECONDARY OBJECTIVES - To assess the safety and tolerability of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age hereditary angioedema (HAE). - To characterize the pharmacokinetics (PK) and pharmacodinamics (PD) of CINRYZE administered by IV injection in children 6 to 11 years of age. - To assess the immunogenecity of CINRYZE following IV administration. OTHER OBJECTIVE - To assess the impact of treatment on health status (quality of life) in children 6 to 11 years of age with HAE.;Primary end point(s): The number of angioedema attacks normalized to a 12-week treatment period.;Timepoint(s) of evaluation of this end point: It will be measured continuously throughout the treatment period. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): SECONDARY EFFICACY ENDPOINTS - Cumulative Attack Severity. This score is the sum of the maximum symptom severity recorded for each angioedema attack in a treatment period. - Cumulative Daily Severity. This score is the sum of the severity scores recorded for every day of reported symptoms in a treatment period. - Time (measured in days from the first dose of study drug in a treatment period) to the first angioedema attack reported in that treatment period. - Change from pre- to post-dose in C1 INH functional activity, C1 INH antigen, and C4 levels. - Number of angioedema attacks requiring acute treatment during each treatment period. SAFETY ENDPOINTS Safety and tolerability, including: - adverse events by dose group - adverse events by exposure (dose normalized to body weight [U/kg] - adverse events by time of onset (e.g. during administration of study drug or within 24 hours after the end of injection of study drug). PK/PD ENDPOINTS Concentrations of C1 INH antigen, functional C1 INH activity, and complement C4 for individual subjects will be determined using validated analytical methods. Results will be summarized using descriptive statistics for values at each time point and for change from baseline at each post-injection time. OTHER ENDPOINTS Results of the EQ-5D-Y health status questionnaire will be presented in accordance with the EQ-5D-3L User Guide (version 4.0) and adaptd as appropriate for the youth version.;Timepoint(s) of evaluation of this end point: •Efficacy endpoints: continuously throughout the treatment period •Safety endpoints: continuously throughout the treatment period The timepoints of blood sampling for determination of the PK/PD endpoints are specified in Schedule 5 on page 22 of the protocol. | — |
Countries
Argentina, Germany, Italy, Mexico, Romania, Spain, United Kingdom, United States
Contacts
Public ContactDanielle Tierens
ViroPharma SPRL
Outcome results
None listed