C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks

This study has been completed.
Sponsor:
Information provided by:
Shire
ClinicalTrials.gov Identifier:
NCT00289211
First received: February 7, 2006
Last updated: March 19, 2014
Last verified: March 2014
Results First Received: March 17, 2010  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Condition: Hereditary Angioedema
Interventions: Biological: C1 esterase inhibitor [human] (C1INH-nf)
Drug: Placebo (saline)

  Participant Flow
  Hide Participant Flow

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
No text entered.

Reporting Groups
  Description
C1INH-nf 1,000 Units (U) of C1 esterase inhibitor (C1INH-nf) administered intravenously (IV). If there was no response to treatment 60 minutes after the first dose, a second 1,000 U dose could be administered.
Placebo Matching placebo (saline) administered IV. If there was no response to treatment 60 minutes after the first dose, a second placebo (saline) dose could be administered.
Open-label C1INH-nf Only Twelve subjects were never randomized but received open-label C1INH-nf for treatment of laryngeal angioedema and/or prior to emergency surgical procedures. These subjects were analyzed for safety only.

Participant Flow:   Overall Study
    C1INH-nf     Placebo     Open-label C1INH-nf Only  
STARTED     36     35     12  
COMPLETED     36     34     2  
NOT COMPLETED     0     1     10  



  Baseline Characteristics
  Hide Baseline Characteristics

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
C1INH-nf 1,000 U of C1INH-nf administered IV. If there was no response to treatment 60 minutes after the first dose, a second 1,000 U dose could be administered.
Placebo Matching placebo (saline) administered IV. If there was no response to treatment 60 minutes after the first dose, a second placebo (saline) dose could be administered.
Open-label C1INH-nf Only Twelve subjects were never randomized but received open-label C1INH-nf for treatment of laryngeal angioedema and/or prior to emergency surgical procedures. These subjects were analyzed for safety only.
Total Total of all reporting groups

Baseline Measures
    C1INH-nf     Placebo     Open-label C1INH-nf Only     Total  
Number of Participants  
[units: participants]
  36     35     12     83  
Age  
[units: years]
Mean ± Standard Deviation
  36.8  ± 17.68     37.0  ± 13.76     36.3  ± 19.42     36.8  ± 16.20  
Gender  
[units: participants]
       
Female     27     28     6     61  
Male     9     7     6     22  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Time to Beginning of Substantial Relief of the Defining Symptom   [ Time Frame: Within 4 hours after initial treatment ]

2.  Secondary:   Number of Subjects With Beginning of Substantial Relief of the Defining Symptom   [ Time Frame: Within 4 hours after initial treatment ]

3.  Secondary:   Time to Complete Resolution of the HAE Attack   [ Time Frame: 72 hours ]

4.  Secondary:   Antigenic C1 Inhibitor (C1INH) Serum Levels   [ Time Frame: Pre-infusion to 1-, 2-, 4-, and 12 hours post-infusion ]

5.  Secondary:   Functional C1INH Serum Levels   [ Time Frame: Pre-infusion to 1-, 2-, 4-, and 12 hours post-infusion ]

6.  Secondary:   Complement C4 Serum Levels   [ Time Frame: Pre-infusion to 1-, 2-, 4-, and 12 hours post-infusion ]


  Serious Adverse Events


  Other Adverse Events


  More Information
  Hide More Information

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: Chief Scientific Officer
Organization: ViroPharma
phone: 610-458-7300


No publications provided by Shire

Publications automatically indexed to this study:

Responsible Party: Chief Scientific Officer, ViroPharma
ClinicalTrials.gov Identifier: NCT00289211     History of Changes
Other Study ID Numbers: LEVP2005-1/Part A
Study First Received: February 7, 2006
Results First Received: March 17, 2010
Last Updated: March 19, 2014
Health Authority: United States: Food and Drug Administration