A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
Condition: Hereditary Angioedema
MeSH terms: Angioedema; Angioedemas, Hereditary
Intervention: C1 inhibitor (human) [C1 INH]
Intervention MeSH terms: Complement C1s; Complement C1 Inhibitor Protein; Complement C1 Inactivator Proteins
Phase: Phase 4
Study type: Interventional
Primary purpose: Prevention
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No

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 (HAE) attack rates.

        3. To assess the immunogenicity of CINRYZE.
    

Eligibility criteria:

        Inclusion Criteria:

        To be eligible for this protocol, subjects must:

          1. Be ≥6 years of age and ≥25 kg body weight.

          2. 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)

          3. 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.

          4. If an adult, be informed of the nature of the study and provide written informed
             consent before any study-specific procedures are performed.

             OR

          5. 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 Criteria:

        To be eligible for this protocol, subjects must not:

          1. 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.

          2. Have a history of abnormal blood clotting or other coagulopathy.

          3. Be taking prescription anticoagulant medication.

          4. Have a history of allergic reaction to CINRYZE or other blood products.

          5. Have participated in any other investigational drug study within the past 30 days
             (other than CINRYZE protocols).

          6. Have received any blood products (other than CINRYZE) within 60 days prior to
             screening.

          7. 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

          8. Be pregnant or breastfeeding.