Clinical trial NCT01756157: Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks
Official title: A Phase 2, Randomized, Double-Blind, Multicenter, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Subcutaneous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema (HAE)
Keywords: Crossover
C1 esterase inhibitor
Prevention
C1 inhibitor
Hereditary Angioedema
Subcutaneous
Recombinant human hyaluronidase
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2012-12-25
Enrollment: 47
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: PREVENTION

Brief summary: The primary objectives of the study are to evaluate the safety, tolerability, and efficacy of two doses of CINRYZE with recombinant human hyaluronidase (rHuPH20) administered by subcutaneous (SC) injection to prevent angioedema attacks.


Eligibility criteria: Inclusion Criteria:

* Be ≥12 years of age.
* Have a confirmed diagnosis of Hereditary Angioedema.

Exclusion Criteria:

* Receipt of any C1 inhibitor (C1 INH) therapy or any blood products for treatment or prevention of an angioedema attack within 7 days before the first dose of study drug.
* Be receiving prophylactic intravenous CINRYZE that exceeds 1000 units every 3 or 4 days (maximum weekly dose 2000 units).
* Have received any androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 7 days prior to the first dose of study drug.
* 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.
* History of allergic reaction to C1 INH products, including CINRYZE or other blood products.
* History of abnormal blood clotting.
* Have a known allergy to hyaluronidase or any other ingredient in the study formulation.

Primary outcomes:
measure: Normalized Number of Angioedema Attacks During the Treatment Period
timeFrame: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Secondary outcomes:
measure: Cumulative Attack-severity During the Treatment Period
timeFrame: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
measure: Cumulative Daily-severity During the Treatment Period
timeFrame: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
measure: Cumulative Symptomatic Days During the Treatment Period
timeFrame: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
measure: Number of Angioedema Attacks Requiring Acute Treatment During the Treatment Period
timeFrame: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: ViroPharma Investigational Site
state: Alabama
city: Scottsdale
country: United States
facility: ViroPharma Investigational Site
state: Arizona
city: Bentonville
country: United States
facility: ViroPharma Investigational Site
state: Arkansas
city: Walnut Creek
country: United States
facility: ViroPharma Investigational Site
state: California
city: Colorado Springs
country: United States
facility: ViroPharma Investigational Site
state: Colorado
city: Tampa
country: United States
facility: ViroPharma Investigational Site
state: Florida
city: Boston
country: United States
facility: ViroPharma Investigational Site
state: Massachusetts
city: Las Vegas
country: United States
facility: ViroPharma Investigational Site
state: Nevada
city: Mineola
country: United States
facility: ViroPharma Investigational Site
state: New York
city: Cincinnati
country: United States
facility: ViroPharma Investigational Site
state: Ohio
city: Columbus
country: United States
facility: ViroPharma Investigational Site
state: Ohio
city: Lake Oswego
country: United States
facility: ViroPharma Investigational Site
state: Oregon
city: Hershey
country: United States
facility: ViroPharma Investigational Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: ViroPharma Investigational Site
state: Pennsylvania
city: Knoxville
country: United States
facility: ViroPharma Investigational Site
state: Tennessee
city: Dallas
country: United States
facility: ViroPharma Investigational Site
state: Texas
city: Spokane
country: United States
facility: ViroPharma Investigational Site
state: Washington
city: Berlin
country: Germany
facility: ViroPharma Investigational Site
city: Essen
country: Germany
facility: ViroPharma Investigational Site
city: Mainz
country: Germany
facility: ViroPharma Investigational Site
city: Munchen
country: Germany
facility: ViroPharma Investigational Site
city: Barcelona
country: Spain
facility: ViroPharma Investigational Site
city: Jonkoping
country: Sweden
facility: ViroPharma Investigational Site
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Riedl MA, Lumry WR, Li HH, Banerji A, Bernstein JA, Ba M, Bjrkander J, Magerl M, Maurer M, Rockich K, Chen H, Schranz J. Subcutaneous administration of human C1 inhibitor with recombinant human hyaluronidase in patients with hereditary angioedema. Allergy Asthma Proc. 2016 Nov;37(6):489-500. doi: 10.2500/aap.2016.37.4006.
citation: Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.