Status:
COMPLETED
A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
Lead Sponsor:
Shire
Conditions:
Hereditary Angioedema
Eligibility:
All Genders
6+ years
Phase:
PHASE4
Brief Summary
The objectives of the study were: 1. To assess the safety and tolerability of escalating doses of CINRYZE. 2. To assess the effect of an escalating dose algorithm for CINRYZE on hereditary angioedema...
Detailed Description
Qualifying subjects entered a 3-step dose escalation algorithm: * Step 1: 1500 Units twice per week (starting dosing regimen for all subjects in the study) * Step 2: 2000 Units twice per week * Step ...
Eligibility Criteria
Inclusion
- To be eligible for this protocol, subjects must:
- Be ≥6 years of age and ≥25 kg body weight.
- Have a confirmed diagnosis of HAE with a documented history of swelling of the face, extremities, gastrointestinal tract, genitalia, or larynx and a history of at least one of the following:
- C1 INH gene mutation
- C4 level below the lower limit of the reference range
- C1 INH antigen level below the lower limit of the reference range
- Functional C1 INH level below the lower limit of the reference range
- Family history of HAE (i.e., grandparent, parent, sibling)
- Have a history of \>1.0 HAE attack per month (average) of any severity during the 3 consecutive months prior to screening while receiving the recommended CINRYZE dosing of 1000 Units every 3 to 4 days via intravenous injection.
- If an adult, be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed.
- OR
- If a child, have a parent/legal guardian who is willing and able to provide written informed consent for the child to participate in the study (with assent from the child when appropriate).
Exclusion
- To be eligible for this protocol, subjects must not:
- Have, as determined by the investigator and/or the sponsor's medical monitor, any surgical or medical condition that could interfere with the administration of study drug or interpretation of study results.
- Have a history of abnormal blood clotting or other coagulopathy.
- Be taking prescription anticoagulant medication.
- Have a history of allergic reaction to CINRYZE or other blood products.
- Have participated in any other investigational drug study within the past 30 days (other than CINRYZE protocols).
- Have received any blood products (other than CINRYZE) within 60 days prior to screening.
- Have any of the following laboratory values at screening:
- Hemoglobin \<8 g/dL
- White blood cell count \<2 x 10\^9/L or \>20 x 10\^9/L
- Platelet count \<50 x 10\^9/L or \>400 x 10\^9/L
- Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \>2.0 x the upper limit of normal
- Blood urea nitrogen and/or creatinine \>2.0 x the upper limit of normal
- Be pregnant or breastfeeding.
Key Trial Info
Start Date :
August 31 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 24 2012
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT00914966
Start Date
August 31 2009
End Date
May 24 2012
Last Update
June 23 2021
Active Locations (11)
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1
Allergy, Asthma and Immunology Associates
Scottsdale, Arizona, United States, 85251
2
Family Allergy and Asthma Center
Atlanta, Georgia, United States, 30342
3
Institute for Asthma and Allergy
Wheaton, Maryland, United States, 20902
4
Winthrop University Hospital
Mineola, New York, United States, 11501