Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

This study has been completed.
Sponsor:
Information provided by:
Dyax Corp.
ClinicalTrials.gov Identifier:
NCT00262080
First received: December 5, 2005
Last updated: April 9, 2010
Last verified: April 2010
Results First Received: December 30, 2009  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Hereditary Angioedema (HAE)
Interventions: Drug: ecallantide
Drug: Phosphate Buffer Saline (PBS),

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients were screened in advance of presenting with an HAE attack but were randomized only upon attack.

Reporting Groups
  Description
Ecallantide / Ecallantide Patients treated with ecallantide in the double-blind part and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Placebo / Ecallantide Patients treated with placebo in the double-blind part and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Ecallantide (Repeat Dose Only) Patients not treated in the double-blind part but treated with ecallantide in the repeat-dosing part.

Participant Flow for 2 periods

Period 1:   Double Blind Treatment Period
    Ecallantide / Ecallantide     Placebo / Ecallantide     Ecallantide (Repeat Dose Only)  
STARTED     36 [1]   36 [2]   0  
COMPLETED     35     36     0  
NOT COMPLETED     1     0     0  
Lost to Follow-up                 1                 0                 0  
[1] One patient was randomized to receive ecallantide but was treated with placebo.
[2] One patient was randomized to receive placebo but was treated with ecallantide.

Period 2:   Repeat Dose Treatment Period
    Ecallantide / Ecallantide     Placebo / Ecallantide     Ecallantide (Repeat Dose Only)  
STARTED     22 [1]   26 [1]   19 [2]
COMPLETED     18 [3]   25     14  
NOT COMPLETED     4     1     5  
Adverse Event                 1                 0                 0  
Withdrawal by Subject                 0                 1                 1  
Lost to Follow-up                 2                 0                 2  
Decision to discontinue the study                 0                 0                 2  
enrolled in EDEMA4                 1                 0                 0  
[1] Not all patients treated in the double-blind part came for treatment in the repeat-dose part.
[2] This arm represents new patients, not treated in the double-blind part of the study.
[3] Some patients participated in only the double-blind or the open label phase.



  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.
No text entered.

Reporting Groups
  Description
Ecallantide / Ecallantide Patients treated with ecallantide in the double-blind part (ITT as treated) and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Placebo / Ecallantide Patients treated with placebo in the double-blind part (ITT as treated) and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Ecallantide (Repeat-Dosing Part Only)

Patients not treated in the double-blind part but treated with ecallantide in the repeat-dosing part.

One patient was omitted from analysis in the intent-to-treat and per-protocol populations due to the loss of the data for the 4-hour post-dose assessments during treatment episode 1.

Total Total of all reporting groups

Baseline Measures
    Ecallantide / Ecallantide     Placebo / Ecallantide     Ecallantide (Repeat-Dosing Part Only)     Total  
Number of Participants  
[units: participants]
  36     36     18     90  
Age  
[units: years]
Mean ± Standard Deviation
  37.1  ± 14.3     33.6  ± 14.6     35.0  ± 14.6     34.7  ± 14.7  
Gender  
[units: participants]
       
Female     23     24     11     58  
Male     13     12     7     32  



  Outcome Measures
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1.  Primary:   Treatment Outcome Score at 4 Hours Post-Dose   [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ]

2.  Secondary:   Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose   [ Time Frame: baseline, 4 hours post-dose (DOUBLE-BLIND PART) ]

3.  Secondary:   Time to Significant Improvement in Overall Response   [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ]

4.  Other Pre-specified:   Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART)   [ Time Frame: Baseline ]

5.  Other Pre-specified:   Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes   [ Time Frame: 4 hours post-dose (REPEAT-DOSING PART) ]

6.  Other Pre-specified:   Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes   [ Time Frame: baseline, 4 hours post-dose (REPEAT-DOSING PART) ]

7.  Other Pre-specified:   Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes   [ Time Frame: 4 hours post-dose (REPEAT-DOSING PART) ]


  Serious Adverse Events


  Other Adverse Events


  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.


Limitations and Caveats
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
No text entered.  


Results Point of Contact:  
Name/Title: Pat T Horn
Organization: Dyax Corp.
phone: 617 250 5948
e-mail: phorn@dyax.com


No publications provided by Dyax Corp.

Publications automatically indexed to this study:

Responsible Party: Bill Pullman, MD, PhD, Executive Vice President, Chief Development Officer, Dyax Corp.
ClinicalTrials.gov Identifier: NCT00262080     History of Changes
Other Study ID Numbers: EDEMA3 (DX-88/14)
Study First Received: December 5, 2005
Results First Received: December 30, 2009
Last Updated: April 9, 2010
Health Authority: United States: Food and Drug Administration