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Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Dyax Corp.
ClinicalTrials.gov Identifier:
NCT01059526
First received: January 28, 2010
Last updated: October 2, 2014
Last verified: October 2014
  Purpose

The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).


Condition Intervention
Hereditary Angioedema
Drug: ecallantide

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Phase 4, Long-Term Observational Safety Study to Evaluate Immunogenicity and Hypersensitivity With Exposure to KALBITOR (Ecallantide) for the Treatment of Acute Attacks of HAE

Resource links provided by NLM:


Further study details as provided by Dyax Corp.:

Primary Outcome Measures:
  • Occurrence of Anaphylaxis or Other Adverse Events Suggestive of Hypersensitivity [ Time Frame: 12 months after first treatment ] [ Designated as safety issue: Yes ]
    Based on medical review of multiple preferred terms for treatment emergent adverse events (TEAEs) suggestive of Type 1 hypersensitivity; terms included adverse drug reaction, anaphylaxis, anaphylactic reaction, anaphylactoid reaction, hypersensitivity, erythema, flushing, hot flush, pharyngeal edema, laryngeal edema, pruritus, pruritus generalized, rash, rash erythematous, rhinitis allergic, rhinorrhea, throat irritation, urticaria, urticaria localized, dyspnea, and wheezing. Records of patients with any of these TEAE referred terms were reviewed further to assess potential hypersensitivity reactions, considering factors such as timing of TEAEs in relationship to dose (ie, occurred within 24 hours after start of KALBITOR treatment), accompanying symptoms, Investigator causality assessment (ie, reported as possibly, probably, or definitely related to study drug), and any other available clinical information. Anaphylaxis subset determined based on criteria established by the NIAID.

  • Occurrence of Seroconversion to Anti-ecallantide Antibodies Upon Exposure to KALBITOR. [ Time Frame: 12 months after first treatment ] [ Designated as safety issue: Yes ]

    Seroconversion is the development of detectable specific antibodies in the blood serum. Serum was tested for development of antibodies (irrespective of immunoglobulin class) against ecallantide at screening and at all safety evaluations. Positive results were to undergo a confirmatory test. Confirmed positive samples were further titered. Patients who developed an antibody response were evaluated for the development of neutralizing antibodies.

    Patients also had their serum analyzed for IgE-specific antibodies to ecallantide at screening and during safety evaluations. Positive results underwent a confirmatory test. Confirmed positive samples were further titered.


  • Occurrence of Adverse Events Related to Disordered Coagulation (Hypercoagulability and Hypocoagulability) Upon Exposure to KALBITOR [ Time Frame: 12 months after first treatment ] [ Designated as safety issue: Yes ]
    Events of ecchymosis, hemorrhage, petechiae, spontaneous hemorrhage, hematoma, gastrointestinal bleeding, hemorrhagic stroke and any other term indicative of a bleeding event or increased tendency for bleeding were reviewed to determine the occurrence of hypocoagulability. Events of clotting, thrombosis, pulmonary embolism, vaso-occlusive stroke, myocardial infarction, and any other term indicative of a clotting event or increased risk of clotting were reviewed to determine the occurrence of hypercoagulability.


Secondary Outcome Measures:
  • Overall Patient Response Assessment [ Time Frame: within 4 hours post dose ] [ Designated as safety issue: No ]
    The Overall Response Assessment was to be completed every 30 minutes after treatment until patient discharge. Patients evaluated their response to treatment as "a lot better or resolved," "a little better," "the same," "a little worse," or "a lot worse." The data presented is based on the best response achieved following a single dose of KALBITOR (Dose A) for the first HAE treatment episode. Responses of "a lot better or resolved" and "a little better" were combined to form a category of "Better." Similarly, "a little worse" and "a lot worse" were combined to form a category of "Worse." Patients treated in a clinic (study site) could have been discharged after an hour and hence may have only had 2 post-treatment evaluations (30 and 60 minutes); response assessments may not have been consistently provided when patients were treated at an alternate site outside of the study site.


Biospecimen Retention:   Samples Without DNA

serum


Enrollment: 81
Study Start Date: February 2010
Study Completion Date: June 2014
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Patients naive to KALBITOR
HAE patients that have not been treated with KALBITOR (ecallantide) prior to enrollment in the study
Drug: ecallantide
30 mg SC
Other Name: Kalbitor
Patients non- naive to KALBITOR
HAE patients that have been treated with KALBITOR prior to enrollment in the study
Drug: ecallantide
30 mg SC
Other Name: Kalbitor

Detailed Description:

The objective of this study is to evaluate immunogenicity and hypersensitivity upon exposure to KALBITOR, in particular:

  1. Determine the rate of anaphylaxis and Type I hypersensitivity reactions upon exposure to KALBITOR.
  2. Determine the rate of seroconversion to anti-ecallantide antibodies upon exposure to KALBITOR.
  3. Determine the rate of adverse events related to disordered coagulation (hypercoagulability and hypocoagulability) upon exposure to KALBITOR.
  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

patients with hereditary angioedema, either naive or non-naive to KALBITOR (ecallantide) prior to enrollment in the study

Criteria

Inclusion Criteria:

  • Patients indicated per the approved product label for KALBITOR
  • Patient or guardian is able to understand and sign the informed consent form
  • Patient is willing and able to undergo a skin test procedure at screening (baseline)

Exclusion Criteria:

  • Patient contraindicated per the approved product label for KALBITOR
  • Patient confirmed pregnancy or active breastfeeding
  • Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01059526

  Show 40 Study Locations
Sponsors and Collaborators
Dyax Corp.
Investigators
Study Director: Yung Chyung, MD Dyax Corp.
  More Information

No publications provided

Responsible Party: Dyax Corp.
ClinicalTrials.gov Identifier: NCT01059526     History of Changes
Other Study ID Numbers: DX-88/24
Study First Received: January 28, 2010
Results First Received: September 10, 2014
Last Updated: October 2, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Dyax Corp.:
HAE

Additional relevant MeSH terms:
Angioedema
Angioedemas, Hereditary
Cardiovascular Diseases
Genetic Diseases, Inborn
Hypersensitivity
Hypersensitivity, Immediate
Immune System Diseases
Skin Diseases
Skin Diseases, Vascular
Urticaria
Vascular Diseases

ClinicalTrials.gov processed this record on November 20, 2014