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A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

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ClinicalTrials.gov Identifier: NCT01912456
Recruitment Status : Completed
First Posted : July 31, 2013
Last Update Posted : July 28, 2017
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Brief Summary:
The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing hereditary angioedema attacks when it is administered under the skin of subjects with hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each subject will enter a run-in period of up to 8-weeks. Subjects who complete the run-in period and who are eligible will then enter the treatment phase which comprises two sequential treatment periods. In the treatment phase, subjects will be randomized to one of four arms consisting of treatment with low- or higher-volume C1-esterase inhibitor in one treatment period and treatment with low- or higher-volume placebo in the other treatment period. The study will measure the number of hereditary angioedema attacks that subjects experience while receiving each treatment.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Types I and II Biological: Low-volume C1-esterase inhibitor Biological: Higher-volume C1-esterase inhibitor Biological: Low-volume placebo Biological: Higher-volume placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: A Double-blind, Randomized, Placebo-controlled, Cross-over Study to Evaluate the Clinical Efficacy and Safety of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
Study Start Date : January 2014
Actual Primary Completion Date : October 2015
Actual Study Completion Date : October 2015


Arm Intervention/treatment
Experimental: Higher-volume placebo, then low-volume C1-esterase inhibitor
A higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks.
Biological: Low-volume C1-esterase inhibitor
Biological: Higher-volume placebo
Experimental: Low-volume C1-esterase inhibitor, then higher-volume placebo
A low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks.
Biological: Low-volume C1-esterase inhibitor
Biological: Higher-volume placebo
Experimental: Low-volume placebo, then higher-volume C1-esterase inhibitor
A low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks then a higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks.
Biological: Higher-volume C1-esterase inhibitor
Biological: Low-volume placebo
Experimental: Higher-volume C1-esterase inhibitor, then low-volume placebo
A higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks.
Biological: Higher-volume C1-esterase inhibitor
Biological: Low-volume placebo



Primary Outcome Measures :
  1. The time-normalized number of hereditary angioedema attacks [ Time Frame: During the treatment phase, up to 32 weeks. ]

Secondary Outcome Measures :
  1. Percentage of subjects with a ≥ 50% reduction in the number of hereditary angioedema attacks [ Time Frame: During the treatment phase, up to 32 weeks. ]
    The percentage of subjects with a ≥ 50% relative reduction in the time-normalized number of hereditary angioedema attacks during treatment with C1-esterase inhibitor compared with placebo

  2. Number of uses of rescue medication [ Time Frame: During the treatment phase, up to 32 weeks. ]
    The time-normalized number of uses of rescue medication during treatment with C1-esterase inhibitor or placebo

  3. Percentage of subjects with adverse events (AEs). [ Time Frame: Within 24 hours of C1-esterase inhibitor or placebo administration. ]
  4. Percentage of subjects with AEs or other specified safety events. [ Time Frame: During the treatment phase, up to 32 weeks. ]
    The percentage of subjects with unsolicited AEs, serious AEs, or other specified safety events during treatment with C1-esterase inhibitor or placebo.

  5. Percentage of subjects experiencing solicited AEs [ Time Frame: During the treatment phase, up to 32 weeks. ]
    The percentage of subjects experiencing solicited AEs (injection site reactions) during treatment with C1-esterase inhibitor or placebo.

  6. Percentage of investigational product injections resulting in solicited AEs [ Time Frame: During the treatment phase, up to 32 weeks. ]
    The percentage of injections of C1-esterase inhibitor or placebo that result in solicited AEs (injection site reactions).



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Run-In Period Inclusion Criteria:

  • Males or females aged 12 years or older.
  • A clinical diagnosis of hereditary angioedema type I or II.
  • Hereditary angioedema attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
  • For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of Screening: use of a stable regimen within 3 months of Screening, with no plans to change.

Eligibility Criteria for Entering Treatment Period 1:

  • Laboratory confirmation of type I or type II hereditary angioedema, including C1-esterase inhibitor functional activity less than 50% AND C4 antigen level below the laboratory reference range.
  • No clinically significant abnormalities as assessed using laboratory parameters.
  • During participation in the run-in period, subjects must have experienced hereditary angioedema attacks that required acute treatment, required medical attention, or caused significant functional impairment.

Exclusion Criteria:

Run-In Period Exclusion Criteria:

  • History of clinical significant arterial or venous thrombosis, or current history of a clinically significant prothrombotic risk.
  • Incurable malignancies at screening.
  • Any clinical condition that will interfere with the evaluation of C1-esterase inhibitor therapy.
  • Clinically significant history of poor response to C1-esterase therapy for the management of hereditary angioedema.
  • Receiving therapy prohibited by the protocol, including medications for hereditary angioedema prophylaxis.
  • Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01912456


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Locations
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United States, Alabama
Study Site
Birmingham, Alabama, United States, 35209
United States, Arizona
Study Site
Scottsdale, Arizona, United States, 85251
United States, California
Study Site
Bell Gardens, California, United States, 90201
Study Site
La Jolla, California, United States, 92093
Study Site
Orange, California, United States, 92868
Study Site
Walnut Creek, California, United States, 94598
United States, Colorado
Study Site
Colorado Springs, Colorado, United States, 80907
United States, Maryland
Study Site
Chevy Chase, Maryland, United States, 20815
United States, Massachusetts
Study Site
Boston, Massachusetts, United States, 02114
United States, Ohio
Study Site
Cincinnati, Ohio, United States, 45267-0563
Study Site
Columbus, Ohio, United States, 43235
Study Site
Toledo, Ohio, United States, 43617
United States, Oklahoma
Study Site
Tulsa, Oklahoma, United States, 74136
United States, Oregon
Study Site
Lake Oswego, Oregon, United States, 97035
United States, Pennsylvania
Study Site
Hershey, Pennsylvania, United States, 17033
United States, Texas
Study Site
Dallas, Texas, United States, 75231
United States, Virginia
Study Site
Richmond, Virginia, United States, 23298
Study Site
Virginia Beach, Virginia, United States, 23452
United States, Washington
Study Site
Spokane, Washington, United States, 99204
Australia, New South Wales
Study Site
Campbelltown, New South Wales, Australia, 2560
Canada, Ontario
Study Site
Hamilton, Ontario, Canada, L8N 3Z5
Study Site
Ottawa, Ontario, Canada, K1Y 4G2
Study Site
Toronto, Ontario, Canada, M4V 1R2
Canada
Study Site
Quebec, Canada, G1V 4M6
Czechia
Study Site
Hradec Kralove, Czechia, 50005
Study Site
Plzen, Czechia, 30460
Hungary
Study Site
Budapest, Hungary, 1125
Israel
Study Site
Tel Aviv, Israel, 64239
Study Site
Tel Hashomer, Israel, 52621
Italy
Study Site
Catania, Italy, 95123
Study Site
Palermo, Italy, 90146
Romania
Study Site
Cluj Napoca, Romania, 400139
Study Site
Mures, Romania, 540103
Spain
Study Site
Barcelona, Spain, 08035
Study Site
Madrid, Spain, 28007
Study Site
Madrid, Spain, 28046
Study Site
Valencia, Spain, 46026
United Kingdom
Study Site
Brighton, United Kingdom, BN2 5BE
Study Site
London, United Kingdom, E1 2ES
Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Global Clinical Program Director CSL Behring

Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01912456     History of Changes
Other Study ID Numbers: CSL830_3001
2013-000916-10 ( EudraCT Number )
First Posted: July 31, 2013    Key Record Dates
Last Update Posted: July 28, 2017
Last Verified: November 2015
Keywords provided by CSL Behring:
Hereditary Angioedema
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement C1s
Complement C1 Inhibitor Protein
Complement C1 Inactivator Proteins
Immunologic Factors
Physiological Effects of Drugs
Complement Inactivating Agents
Immunosuppressive Agents