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Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

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ClinicalTrials.gov Identifier: NCT02584959
Recruitment Status : Completed
First Posted : October 23, 2015
Last Update Posted : September 6, 2017
Sponsor:
Information provided by (Responsible Party):
Shire

Study Description
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Brief Summary:
The purpose of this study is to assess the efficacy and safety of subcutaneous administration of a liquid formulation of C1 esterase inhibitor for the prevention of angioedema attacks in adolescent and adult subjects with hereditary angioedema.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema (HAE) Drug: C1 esterase inhibitor [human] liquid Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 66 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Prevention
Official Title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Two-period, Three-sequence, Partial Crossover Study to Evaluate the Efficacy and Safety of Subcutaneous Administration of 2000 IU of C1 Esterase Inhibitor [Human] Liquid for Injection for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
Actual Study Start Date : November 1, 2015
Actual Primary Completion Date : July 24, 2017
Actual Study Completion Date : July 24, 2017

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Experimental/Placebo
Subjects will be randomized to receive C1 Esterase Inhibitor in the 1st Treatment period and then switch to Placebo in the 2nd treatment period.
 Drug: C1 esterase inhibitor [human] liquid
C1 Esterase Inhibitor [Human] Liquid administered Subcutaneously as specified on specified days

Drug: Placebo
Placebo
Experimental: Placebo/Experimental
Subjects will be randomized to receive a placebo treatment in the 1st Treatment period and then switch to receive C1 Esterase Inhibitor in the 2nd treatment period.
 Drug: C1 esterase inhibitor [human] liquid
C1 Esterase Inhibitor [Human] Liquid administered Subcutaneously as specified on specified days

Drug: Placebo
Placebo
Experimental: Experimental/ Experimental
Subjects will be randomized and receive C1 Esterase Inhibitor in both 1st as well as the 2nd treatment period
 Drug: C1 esterase inhibitor [human] liquid
C1 Esterase Inhibitor [Human] Liquid administered Subcutaneously as specified on specified days


Outcome Measures
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Primary Outcome Measures :
  1. Time-Normalized Number of Attacks (NNA) for participants [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]

Secondary Outcome Measures :
  1. Proportion of subjects meeting at least a 50% reduction in the NNA during the experimental injection treatment period relative to the placebo period. [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  2. Cumulative attack severity [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  3. Number of attack-free days [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  4. Number of angioedema attacks requiring acute treatment [ Time Frame: Each treatment period: Weeks 1 to 14 ]
  5. Response to icatibant when administered for an acute attack [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  6. Number of subjects with adverse events (AEs) [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]
  7. Number of patients with injection site reactions [ Time Frame: Weeks 1 to 14 for treatment period 1 and 2 ]

Eligibility Criteria
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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
The maximum duration of participation is approximately 9 months. Patients will complete a screening period of up to 21 days. Following screening, eligible patients will be randomly assigned to 1 of 3 treatment sequences. Each patient will undergo 2 14-week treatment periods for a total of 28 weeks (Treatment Period 1 and Treatment Period 2). After completing the 2 treatment periods, patients will enter a 1-month follow-up period.
Contacts and Locations
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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): NCT02584959


  Show 27 Study Locations
Sponsors and Collaborators
Shire
Investigators
Study Director: Shire Physician Shire
More Information
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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02584959     History of Changes
Other Study ID Numbers: SHP616-300
First Posted: October 23, 2015    Key Record Dates
Last Update Posted: September 6, 2017
Last Verified: September 2017

Additional relevant MeSH terms:
Angioedema
Angioedemas, Hereditary
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