Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
Condition: Hereditary Angioedema (HAE)
MeSH terms: Angioedema; Angioedemas, Hereditary
Intervention: CINRYZE 500 U; CINRYZE 1000 U
Intervention description: IV infusion administered twice weekly; IV infusion administered twice weekly
Intervention MeSH terms: Complement C1 Inhibitor Protein
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 8.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to determine if an investigational treatment is safe and well
      tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Be of Japanese descent, defined as born in Japan and having Japanese parents and
             Japanese maternal and paternal grandparents.

          2. Be ≥2 years of age.

          3. Meet the following minimum body weight criteria:

               -  Subjects 2 to 5 years of age must weigh at least 12.5 kg; and

               -  Subjects 6 years of age and above must weigh at least 25 kg.

          4. Have a confirmed diagnosis of Type I or Type II HAE. NOTE: Diagnosis may be based on
             historical data including family history, clinical symptoms (characteristic attacks),
             or documentation of low level of C1 INH protein and/or C1 INH activity.

          5. Have a history of at least one angioedema attack per month (on average) during the 3
             consecutive months immediately before enrollment.

          6. Agree to adhere to the protocol-defined schedule of assessments and procedures.

          7. Agree to avoid his/her known angioedema attack triggers during the study to the best
             of his/her ability.

          8. If a female of reproductive age, be postmenopausal (≥12 months following cessation of
             menstruation), surgically sterile, or following an acceptable method of birth control
             (and agree to continue its use through 1 month after the last dose of study drug):

               -  Non-hormonal methods (eg, abstinence, barrier control) for at least 1 complete
                  menstrual cycle before the Screening Visit.

               -  Stable doses of estrogen and/or progestin containing products for at least 2
                  months before the Screening Visit.

          9. If a male of reproductive age, be surgically sterile or agree to follow an acceptable
             method of birth control (eg, abstinence, barrier control) from the Screening Visit
             through 2 months after the last dose of study drug.

         10. 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 or minor (<20 years of age), have a parent/legal guardian who is informed of
        the nature of the study provide written informed consent (ie, permission) for the child to
        participate in the study before any study-specific procedures are performed. Assent will be
        obtained from children ≥14 years of age.

        Exclusion Criteria:

          1. Have a history of hypercoagulability (abnormal blood clotting).

          2. Have a diagnosis of acquired angioedema or be known to have C1 INH antibodies.

          3. Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of
             the components of CINRYZE) or other blood products.

          4. Have received C1 INH therapy or any blood products within 3 days before the first dose
             of study drug.

          5. Have had signs or symptoms of an angioedema attack within 2 days before the first dose
             of study drug.

          6. Have any change (start, stop, or change in dose) in androgen therapy (eg, danazol,
             oxandrolone, stanozolol, testosterone), tranexamic acid, epsilon-aminocaproic acid
             (EACA), or other antifibrinolytics within 14 days before the first dose of study drug.

          7. If female, have started taking or changed the dose of any hormonal contraceptive
             regimen or hormone replacement therapy (eg, estrogen/progestin containing products)
             within 2 months before the first dose of study drug.

          8. Be pregnant or breastfeeding.

          9. Have received an investigational drug other than those required for prevention or
             treatment of angioedema attacks within 30 days before the first dose of study drug.

         10. 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.