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

This study has been completed.
Sponsor:
Information provided by:
Shire
ClinicalTrials.gov Identifier:
NCT01005888
First received: October 29, 2009
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: Crossover Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Prevention
Condition: Hereditary Angioedema
Interventions: Biological: C1 esterase inhibitor [human] (C1INH-nf)
Drug: Placebo (saline)

  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
A total of 26 subjects were enrolled in the study (see Detailed Description). One subject received open-label C1 esterase inhibitor (C1INH-nf) but withdrew prior to randomization. Another subject was randomized but withdrew prior to receiving study drug. 24 subjects were randomized and began therapy with blinded study drug in Period 1.

Reporting Groups
  Description
C1INH-nf First, Then Placebo 1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks.
Placebo First, Then C1INH-nf Matching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.

Participant Flow for 2 periods

Period 1:   First Intervention
    C1INH-nf First, Then Placebo     Placebo First, Then C1INH-nf  
STARTED     12     12  
COMPLETED     11     11  
NOT COMPLETED     1     1  

Period 2:   Second Intervention
    C1INH-nf First, Then Placebo     Placebo First, Then C1INH-nf  
STARTED     11     11  
COMPLETED     10     10  
NOT COMPLETED     1     1  



  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
C1INH-nf First, Then Placebo 1,000 U of C1INH-nf administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks.
Placebo First, Then C1INH-nf Matching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.
Open-label C1INH-nf Only One subject received open-label C1INH-nf but withdrew prior to randomization.
Randomized, Not Treated One subject was randomized but withdrew prior to receiving study drug.
Total Total of all reporting groups

Baseline Measures
    C1INH-nf First, Then Placebo     Placebo First, Then C1INH-nf     Open-label C1INH-nf Only     Randomized, Not Treated     Total  
Number of Participants  
[units: participants]
  12     12     1     1     26  
Age  
[units: participants]
         
<=18 years     2     2     0     1     5  
Between 18 and 65 years     9     10     1     0     20  
>=65 years     1     0     0     0     1  
Gender  
[units: participants]
         
Female     9     12     1     0     22  
Male     3     0     0     1     4  



  Outcome Measures
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1.  Primary:   Number of Hereditary Angioedema (HAE) Attacks During Each Prophylactic Therapy Period   [ Time Frame: 12 weeks ]

2.  Secondary:   Number of Subject Withdrawals During Each Prophylactic Therapy Period   [ Time Frame: 12 weeks ]

3.  Secondary:   Average Severity of HAE Attacks During Each Prophylactic Therapy Period   [ Time Frame: 12 weeks ]

4.  Secondary:   Average Duration of HAE Attacks During Each Prophylactic Therapy Period   [ Time Frame: 12 weeks ]

5.  Secondary:   Number of Open-label C1INH-nf Infusions Required During Each Prophylactic Therapy Period   [ Time Frame: 12 weeks ]

6.  Secondary:   Antigenic C1 Inhibitor (C1INH) Serum Levels   [ Time Frame: Pre-infusion to 1 hour post-infusion at Visit 1 and Weeks 4, 8, and 12 ]

7.  Secondary:   Functional C1INH Serum Levels   [ Time Frame: Pre-infusion to 1 hour post-infusion at Visit 1 and Weeks 4, 8, and 12 ]

8.  Other Pre-specified:   Total Number of Days of Swelling During Each Prophylactic Therapy Period   [ Time Frame: 12 weeks ]


  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
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  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: 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: NCT01005888     History of Changes
Other Study ID Numbers: LEVP2005-1/Part B
Study First Received: October 29, 2009
Results First Received: March 17, 2010
Last Updated: March 19, 2014
Health Authority: United States: Food and Drug Administration