A PHASE 3, MULTICENTER, RANDOMIZED, SINGLE-BLIND, DOSE-RANGING, CROSSOVER STUDY TO EVALUATE THE SAFETY AND EFFICACY OF INTRAVENOUS ADMINISTRATION OF CINRYZE® (C1 ESTERASE INHIBITOR [HUMAN]) FOR THE PREVENTION OF ANGIOEDEMA ATTACKS IN CHILDREN 6 TO 11 YEARS OF AGE WITH HEREDITARY ANGIOEDEMA

Phase 1

• Conditions: Prevention of angioedema attacks in children 6 to 11 years of age with hereditary angioedema.; MedDRA version: 16.1Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2013-002453-29-IT
• Lead Sponsor: ViroPharma Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-01-22
• Study Completion: 2017-05-04
• Last Update Posted: 9 October 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

1. Be a child (male of female), =6 to < 12years of age at the time of screening.
2. Have a confirmed diagnosis of Type I or Type II HAE and have a functional C1 inhibitor (C1 INH) level less than 50% of normal.
3. Have a history of = 1.0 moderate or severe angioedema attacks per month (average) during the 3 consecutive months prior to screening.
4. Agree to adhere to the protocol-defined schedule of assessments and procedures.
5. Have a parent(s)/legal guardian who is informed of the nature of the study provide written informed consent for the child to participate in the study before any study-specific procedures are performed (with assent from the child when appropriate).
Additional Inclusion Criteria (Qualifying for Randomization):
6. Have experienced = 1.0 moderate or severe angioedema attacks per month (average) during the 12-week baseline observation period.

Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>- 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).<br>- To characterize the pharmacokinetics (PK) and pharmacodinamics (PD) of CINRYZE administered by IV injection in children 6 to 11 years of age.<br>- To assess the immunogenecity of CINRYZE following IV administration.<br><br>OTHER OBJECTIVE<br>- 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.