CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
- Conditions
- Hereditary Angioedema (HAE)
- Interventions
- Biological: CINRYZE
- Registration Number
- NCT01095510
- Lead Sponsor
- Shire
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 9
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.
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description 1500 U CINRYZE (>25 kg body weight) CINRYZE Single IV dose of 1500 U CINRYZE 1000 U CINRYZE (10-25 kg body weight) CINRYZE Single IV dose of 1000 U CINRYZE 500 U CINRYZE (10-25 kg body weight) CINRYZE Single IV dose of 500 U CINRYZE 1000 U CINRYZE (>25 kg body weight) CINRYZE Single IV dose of 1000 U CINRYZE
- Primary Outcome Measures
Name Time Method Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom Within 4 hours following treatment
- Secondary Outcome Measures
Name Time Method 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 Data was not reported due to change in planned analysis.
Time to Unequivocal Beginning of Relief of the Defining Attack Symptom Within 4 hours following treatment Time to Complete Resolution of the Attack Within 1 week following treatment
Trial Locations
- Locations (11)
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
🇺🇸Tampa, Florida, United States
Allergy & Asthma Research Group
🇺🇸Eugene, Oregon, United States
Baker Allergy, Asthma and Dermatology Research Center, LLC
🇺🇸Lake Oswego, Oregon, United States
Marycliff Allergy Specialists
🇺🇸Spokane, Washington, United States
Klinikum rechts der Isar, Technical University Munich, ENT Clinic
🇩🇪Munich, Germany
Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
🇩🇪Berlin, Germany
Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center
🇭🇺Budapest, Hungary
AARA Research Center
🇺🇸Dallas, Texas, United States
Allergy and Asthma Research Center, P.A.
🇺🇸San Antonio, Texas, United States
Asthma & Allergy Associates, P.C.
🇺🇸Colorado Springs, Colorado, United States
Institute for Asthma and Allergy, PC
🇺🇸Chevy Chase, Maryland, United States