CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
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
2 other identifiers
interventional
9
3 countries
11
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2010
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 24, 2010
CompletedFirst Posted
Study publicly available on registry
March 30, 2010
CompletedStudy Start
First participant enrolled
June 2, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 17, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
April 17, 2012
CompletedResults Posted
Study results publicly available
July 25, 2014
CompletedJune 3, 2021
May 1, 2021
1.9 years
March 24, 2010
June 26, 2014
May 11, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Within 4 hours following treatment
Secondary Outcomes (3)
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
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
Study Arms (4)
500 U CINRYZE (10-25 kg body weight)
EXPERIMENTALSingle IV dose of 500 U CINRYZE
1000 U CINRYZE (10-25 kg body weight)
EXPERIMENTALSingle IV dose of 1000 U CINRYZE
1000 U CINRYZE (>25 kg body weight)
EXPERIMENTALSingle IV dose of 1000 U CINRYZE
1500 U CINRYZE (>25 kg body weight)
EXPERIMENTALSingle IV dose of 1500 U CINRYZE
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (11)
Asthma & Allergy Associates, P.C.
Colorado Springs, Colorado, 80907, United States
University of South Florida Asthma, Allergy and Immunology Clinical Research Unit
Tampa, Florida, 33613, United States
Institute for Asthma and Allergy, PC
Chevy Chase, Maryland, 20815, United States
Allergy & Asthma Research Group
Eugene, Oregon, 97401, United States
Baker Allergy, Asthma and Dermatology Research Center, LLC
Lake Oswego, Oregon, 97035, United States
AARA Research Center
Dallas, Texas, 75231, United States
Allergy and Asthma Research Center, P.A.
San Antonio, Texas, 78229, United States
Marycliff Allergy Specialists
Spokane, Washington, 99204, United States
Charité Universitätsmedizin Berlin, Dept. of Dermatology and Allergy
Berlin, Germany
Klinikum rechts der Isar, Technical University Munich, ENT Clinic
Munich, Germany
Semmelweis University, Allergy and Angioedema Outpatients Clinic, Kútvölgyi Clinical Center
Budapest, Hungary
Related Publications (1)
Lumry W, Soteres D, Gower R, Jacobson KW, Li HH, Chen H, Schranz J. Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema. Pediatr Allergy Immunol. 2015 Nov;26(7):674-80. doi: 10.1111/pai.12444. Epub 2015 Aug 11.
PMID: 26171584RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Change in C1 INH antigen and functional C1 INH concentrations endpoint was not analyzed as no participants agreed to additional and optional blood sampling. As a result, no PK parameters were calculated for this study.
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 24, 2010
First Posted
March 30, 2010
Study Start
June 2, 2010
Primary Completion
April 17, 2012
Study Completion
April 17, 2012
Last Updated
June 3, 2021
Results First Posted
July 25, 2014
Record last verified: 2021-05