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: 17.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-12-24
• Study Completion: 2017-05-04
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• 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 angioedema attacks per month (average) that are moderate or severe or require acute treatment 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 angioedema attacks per month (average) that are moderate or severe or require acute treatment 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