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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
Results First Received: September 10, 2014  
Study Type: Observational
Study Design: Observational Model: Cohort;   Time Perspective: Prospective
Condition: Hereditary Angioedema
Intervention: Drug: ecallantide

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients indicated in the US FDA-approved product label for KALBITOR (ecallantide) who experienced an acute attack of HAE were eligible for inclusion in this trial.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Subjects were to complete screening/enrollment procedures on a separate day prior to receiving KALBITOR treatment for an acute attack of HAE. The primary and secondary endpoints were analyzed for the Safety population only (all patients who received at least 1 treatment dose of KALBITOR).

Reporting Groups
  Description
Patients Naive to KALBITOR

HAE patients that have not been treated with KALBITOR (ecallantide) prior to enrollment in the study

ecallantide: 30 mg SC

Patients Non- Naive to KALBITOR

HAE patients that have been treated with KALBITOR prior to enrollment in the study

ecallantide: 30 mg SC


Participant Flow:   Overall Study
    Patients Naive to KALBITOR     Patients Non- Naive to KALBITOR  
STARTED     41 [1]   40 [1]
Treated     21 [2]   23 [2]
COMPLETED     11     18  
NOT COMPLETED     30     22  
Patient enrolled but not treated                 20                 17  
Adverse Event                 2                 2  
Protocol Violation                 0                 1  
Withdrawal by Subject                 3                 1  
Lost to Follow-up                 4                 1  
Patient found to be dermatographic                 1                 0  
[1] ITT population; defined as signing informed consent and meeting all inclusion/ exclusion criteria
[2] Safety population; defined as all patients who received at least 1 treatment dose of KALBITOR



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety population; defined as all patients who received at least 1 treatment dose of KALBITOR

Reporting Groups
  Description
Patients Naive to KALBITOR

HAE patients that have not been treated with KALBITOR (ecallantide) prior to enrollment in the study

ecallantide: 30 mg SC

Patients Non- Naive to KALBITOR

HAE patients that have been treated with KALBITOR prior to enrollment in the study

ecallantide: 30 mg SC

Total Total of all reporting groups

Baseline Measures
    Patients Naive to KALBITOR     Patients Non- Naive to KALBITOR     Total  
Number of Participants  
[units: participants]
  21     23     44  
Age  
[units: years]
Mean ± Standard Deviation
  48.2  ± 16.09     38.3  ± 14.86     43.0  ± 16.08  
Gender  
[units: participants]
     
Female     15     15     30  
Male     6     8     14  
Race/Ethnicity, Customized  
[units: participants]
     
Caucasian     20     22     42  
Black     1     1     2  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Occurrence of Anaphylaxis or Other Adverse Events Suggestive of Hypersensitivity   [ Time Frame: 12 months after first treatment ]

2.  Primary:   Occurrence of Seroconversion to Anti-ecallantide Antibodies Upon Exposure to KALBITOR.   [ Time Frame: 12 months after first treatment ]

3.  Primary:   Occurrence of Adverse Events Related to Disordered Coagulation (Hypercoagulability and Hypocoagulability) Upon Exposure to KALBITOR   [ Time Frame: 12 months after first treatment ]

4.  Secondary:   Overall Patient Response Assessment   [ Time Frame: within 4 hours post dose ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
44 patients (out of 200 planned) received treatment in this study. Additionally, the collection of evaluable efficacy data for the secondary endpoint was limited by a lack of response data collected for HAE attacks treated at alternate sites.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Burt Adelman, MD
Organization: Dyax Corp.
phone: 617-225-2500
e-mail: badelman@dyax.com


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