Clinical trial NCT01095497: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
Official title: An Open-Label Multiple-Dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous (SC) Versus Intravenous (IV) Administration of CINRYZE in Adolescents and Adults With Hereditary Angioedema (HAE)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema
Keywords: HAE
C1 inhibitor (C1INH)
Subcutaneous
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2010-03-30
Enrollment: 26
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: OTHER

Brief summary: The objectives of the study are to:

1. Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema
2. Characterize the pharmacokinetics and pharmacodynamics of CINRYZE administered by subcutaneous injection
3. Assess the immunogenicity of CINRYZE following subcutaneous administration


Eligibility criteria: Inclusion Criteria:

To be eligible for this protocol, a subject must:

1. Have a confirmed diagnosis of HAE.
2. During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) treated with C1INH therapy or any other blood products, ecallantide (Kalbitor), icatibant (Firazyr), antifibrinolytics (e.g., tranexamic acid), IV fluids, or narcotic analgesics.
3. Agree to strictly adhere to the protocol-defined schedule of assessments and procedures.

Exclusion Criteria:

To be eligible for this protocol, a subject must not:

1. Have received C1INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose.
2. Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose.
3. Have any change (start, stop, or change in dose) in androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 14 days prior to the first dose.
4. If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose.
5. Have a history of abnormal blood clotting or other coagulopathy.
6. Have a history of allergic reaction to CINRYZE or other blood products.
7. Be pregnant or breastfeeding.
8. Have received an immunization within 30 days prior to the first dose.
9. Have participated in any other investigational drug study within 30 days prior to the first dose.

Primary outcomes:
measure: Incidence and Severity of Adverse Events, Number of Subjects With Local Injection Site Reactions, and Number of Subjects Who Discontinue Study Drug or Withdraw From the Study.
timeFrame: 18 days in each treatment period
Secondary outcomes:
measure: Mean Change C1 Inhibitor (C1INH)
timeFrame: 18 days in each treatment period
measure: Mean Change C4 Compliment
timeFrame: 18 days in each treatment period
measure: Number of Participants With C1 Inhibitor (INH) Antibodies
timeFrame: 18 days in each treatment period
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Allergy, Asthma and Immunology Associates
state: Arizona
city: Walnut Creek
country: United States
facility: Allergy and Asthma Clinical Research, Inc.
state: California
city: Atlanta
country: United States
facility: Family Allergy and Asthma Center
state: Georgia
city: Chevy Chase
country: United States
facility: Institute for Asthma and Allergy
state: Maryland
city: Lake Oswego
country: United States
facility: Allergy, Asthma and Dermatology Research Center
state: Oregon
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Spokane
country: United States
facility: Marycliff Allergy Specialists
state: Washington
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: