Clinical trial NCT01426763: A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase
Official title: An Open-label Multiple-dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Hereditary Angioedema
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema
Keywords: HAE
C1 esterase inhibitor
C1 INH
Subcutaneous
hyaluronidase
rHuPH20
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Halozyme Therapeutics
First posted: 2011-08-31
Enrollment: 12
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: The objectives of the study are to:

1. Evaluate the safety and tolerability of subcutaneously administered CINRYZE with recombinant human hyaluronidase (rHuPH20) in subjects with hereditary angioedema (HAE) who previously participated in CINRYZE Study 0624-200 (NCT01095497)
2. Characterize the pharmacokinetics and pharmacodynamics of subcutaneously administered CINRYZE with rHuPH20
3. Assess the immunogenicity of CINRYZE following subcutaneous (SC) administration of CINRYZE with rHuPH20


Eligibility criteria: Inclusion Criteria:

To be eligible for this protocol, a subject must:

1. Provide informed consent/assent, as appropriate.
2. Have previously participated in CINRYZE Study 0624-200 and completed the subcutaneous therapy period in that study.
3. During the 3 consecutive months prior to screening, have a history of less than 1 HAE attack per month (average) that required treatment with C1 INH therapy or other blood products.
4. Agree to avoid his/her known HAE triggers during the study to the best of his/her ability.

Exclusion Criteria:

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

1. Have received C1 INH therapy or any blood products for treatment or prevention of an HAE attack within 14 days prior to the first dose of study drug.
2. Have received any ecallantide (Kalbitor), icatibant (Firazyr), or antifibrinolytics (e.g., tranexamic acid) within 14 days prior to the first dose of study drug.
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 of study drug.
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 of study drug.
5. Have a history of abnormal blood clotting.
6. Have a history of allergic reaction to products containing C1 INH or other blood products.
7. Have a known allergy to hyaluronidase or any other ingredient in rHuPH20.
8. Be pregnant or breastfeeding.
9. Have received an investigational study drug within 30 days prior to the first dose of study drug.

Primary outcomes:
measure: Incidence and Severity of Adverse Events, Number of Participants With Local Injection Site Reactions, and Number of Participants Who Discontinue Study Drug or Withdraw From the Study
timeFrame: 18 days
Secondary outcomes:
measure: Mean Change C1 Inhibitor (C1INH)
timeFrame: 18 days
measure: Mean Change C4 Compliment
timeFrame: 18 days
measure: Number of Subjects With C1 INH Antibodies
timeFrame: Day 1 (pre-dose), Day 18 (168 h post Dose 4), and 30 (±2) days after the last dose of study drug (Dose 4)
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: ViroPharma Investigational Site
state: Arizona
city: Walnut Creek
country: United States
facility: ViroPharma Investigational Site
state: California
city: Atlanta
country: United States
facility: ViroPharma Investigational Site
state: Georgia
city: Dallas
country: United States
facility: ViroPharma Investigational Site
state: Texas
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: