CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

Phase 2

Completed

• Conditions: Hereditary Angioedema (HAE)

• 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.

Study Timeline

• Study First Submitted: 2010-03-24
• Study First Submitted QC: 2010-03-26
• Study First Posted: 2010-03-30
• Study Start: 2010-06-02
• Primary Completion: 2012-04-17
• Study Completion: 2012-04-17
• Results First Submitted: 2014-06-26
• Results First Submitted QC: 2014-06-26
• Results First Posted: 2014-07-25
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-11
• Last Update Posted: 2021-06-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

To be eligible for this protocol, subjects must:

1. Be at least 10 kg of body weight.
2. Have a confirmed diagnosis of HAE.
3. 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:

1. Have any active infectious illness.
2. Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
3. 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.
4. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
5. 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

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)

Asthma & Allergy Associates, P.C.; 🇺🇸 Colorado Springs, Colorado, United States

University of South Florida Asthma, Allergy and Immunology Clinical Research Unit; 🇺🇸 Tampa, Florida, United States

Institute for Asthma and Allergy, PC; 🇺🇸 Chevy Chase, Maryland, United States

Allergy & Asthma Research Group; 🇺🇸 Eugene, Oregon, United States

Baker Allergy, Asthma and Dermatology Research Center, LLC; 🇺🇸 Lake Oswego, Oregon, United States

AARA Research Center; 🇺🇸 Dallas, Texas, United States

Allergy and Asthma Research Center, P.A.; 🇺🇸 San Antonio, Texas, United States

Marycliff Allergy Specialists; 🇺🇸 Spokane, Washington, United States

Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy; 🇩🇪 Berlin, Germany

Klinikum rechts der Isar, Technical University Munich, ENT Clinic; 🇩🇪 Munich, Germany

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