Open-Label, Single-Dose Study to Evaluate the Response and Pharmacokinetics/Pharmacodynamics of Different Doses of CINRYZE® [C1 Inhibitor (Human)] For Treatment of Acute Angioedema Attacks in Children Less Than 12 Years of Age With Hereditary Angioedema

Shire11 sites in 3 countries9 target enrollmentJune 2, 2010

ConditionsHereditary Angioedema (HAE)

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Hereditary Angioedema (HAE)
Sponsor: Shire
Enrollment: 9
Locations: 11
Primary Endpoint: Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Status: Completed
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.

Detailed Description

Each subject received CINRYZE for treatment of a single acute angioedema attack.

Registry

clinicaltrials.gov

Start Date

June 2, 2010

End Date

April 17, 2012

Last Updated

4 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Shire

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•To be eligible for this protocol, subjects must:
•Be at least 10 kg of body weight.
•Have a confirmed diagnosis of HAE.
•Have an acute HAE attack and be able to initiate treatment within 8 hours after onset of symptoms.

Exclusion Criteria

•To be eligible for this protocol, subjects must not:
•Have any active infectious illness.
•Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
•Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
•Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
•Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with CINRYZE in this study at any time.

Outcomes

Primary Outcomes

Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom

Time Frame: Within 4 hours following treatment

Secondary Outcomes

Time to Unequivocal Beginning of Relief of the Defining Attack Symptom(Within 4 hours following treatment)
Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations(Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8)
Time to Complete Resolution of the Attack(Within 1 week following treatment)