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Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 3)

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ClinicalTrials.gov Identifier: NCT00462709
Recruitment Status : Completed
First Posted : April 19, 2007
Results First Posted : May 3, 2010
Last Update Posted : April 15, 2014
Sponsor:
Information provided by:
Shire

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Condition Hereditary Angioedema
Intervention Biological: C1 esterase inhibitor [human] (C1INH-nf)
Enrollment 146

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description 1,000 Units (U) of C1 esterase inhibitor (C1INH-nf) administered intravenously (IV) every 3 to 7 days.
Period Title: Overall Study
Started 146
Completed 79
Not Completed 67
Reason Not Completed
Transitioned to commercial C1INH-nf             40
Lost to Follow-up             10
Withdrawal by Subject             8
Transferred to LEVP2006-1 (NCT00438815)             3
Death             2
Logistical reasons             2
Physician Decision             1
Withdrew to start treatment with Cetor             1
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description 1,000 U of C1INH-nf administered IV every 3 to 7 days.
Overall Number of Baseline Participants 146
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 146 participants
36.5  (16.48)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 146 participants
Female
112
  76.7%
Male
34
  23.3%
1.Primary Outcome
Title Frequency of All HAE Attacks
Hide Description A hereditary angioedema (HAE) attack was defined as a discrete episode during which the subject progressed from no angioedema to symptoms of angioedema.
Time Frame Duration of the study
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat Efficacy (ITT-E) Population (N=146; the number of subjects who received at least one prophylactic dose of C1INH-nf for the prevention of HAE attacks). HAE attack frequency was reported by 137 subjects at screening (i.e., data were missing for 9 subjects).
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description:
1,000 U of C1INH-nf administered IV every 3 to 7 days.
Overall Number of Participants Analyzed 146
Median (Full Range)
Unit of Measure: attacks per month
At Screening (N=137)
3.00
(0.08 to 28.00)
During Prophylactic Therapy with C1INH-nf (N=146)
0.21
(0.00 to 4.56)
2.Other Pre-specified Outcome
Title Antigenic C1 Inhibitor (C1INH) Serum Levels
Hide Description Change from pre-infusion to 1 hour post-infusion in antigenic C1INH serum levels.
Time Frame Pre-infusion to 1 hour post-infusion
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT-E subjects with data at both sampling time points (N=137).
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description:
1,000 U of C1INH-nf administered IV every 3 to 7 days.
Overall Number of Participants Analyzed 137
Mean (Standard Deviation)
Unit of Measure: mg/dL
Pre-infusion 17.1  (19.67)
Increase at 1 hour post-infusion 8.6  (11.89)
3.Other Pre-specified Outcome
Title Functional C1INH Serum Levels
Hide Description

Change from pre-infusion to 1 hour post-infusion in functional C1INH serum levels.

Functional C1INH serum levels are expressed as a percent of total detectable C1INH (i.e., functional C1INH/total detectable C1INH).

Time Frame Pre-infusion to 1 hour post-infusion
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT-E subjects with data at both sampling time points (N=132).
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description:
1,000 U of C1INH-nf administered IV every 3 to 7 days.
Overall Number of Participants Analyzed 132
Mean (Standard Deviation)
Unit of Measure: percent
Pre-infusion 42.0  (25.85)
Percent increase at 1 hour post-infusion 30.4  (18.05)
4.Other Pre-specified Outcome
Title Complement C4 Serum Levels
Hide Description Change from pre-infusion to 1 hour post-infusion in complement C4 serum levels.
Time Frame Pre-infusion to 1 hour post-infusion
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT-E subjects with data at both sampling time points (N=134).
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description:
1,000 U of C1INH-nf administered IV every 3 to 7 days.
Overall Number of Participants Analyzed 134
Mean (Standard Deviation)
Unit of Measure: mg/dL
Pre-infusion 10.1  (8.17)
Change at 1 hour post-infusion -0.1  (3.61)
Time Frame [Not Specified]
Adverse Event Reporting Description Presented are treatment-emergent adverse reactions considered to be related to C1INH-nf. There were no serious adverse reactions considered related to C1INH-nf.
 
Arm/Group Title Open-label C1INH-nf
Hide Arm/Group Description 1,000 U of C1INH-nf administered IV every 3 to 7 days.
All-Cause Mortality
Open-label C1INH-nf
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Open-label C1INH-nf
Affected / at Risk (%)
Total   0/146 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Open-label C1INH-nf
Affected / at Risk (%)
Total   9/146 (6.16%) 
Gastrointestinal disorders   
Nausea  2/146 (1.37%) 
Nervous system disorders   
Headache  3/146 (2.05%) 
Skin and subcutaneous tissue disorders   
Rash  3/146 (2.05%) 
Vascular disorders   
Phlebitis  2/146 (1.37%) 
1
Term from vocabulary, MedDRA (9.0)
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.
Clinical Study Agreement. Most restrictive provision - PI will not publish results until after first of: multicenter publication is published or 24 months from study end. Thereafter, PI may publish his results. PI must provide copy of proposed publication to sponsor for pre-review. If sponsor requests, PI must delete sponsor confidential information before publication and/or delay publication for 90 days so sponsor can file for patents or take other action to protect its patent rights.
Results Point of Contact
Name/Title: Chief Scientific Officer
Organization: ViroPharma
Phone: 610-458-7300
Responsible Party: Chief Scientific Officer, ViroPharma
ClinicalTrials.gov Identifier: NCT00462709     History of Changes
Other Study ID Numbers: LEVP2006-4
First Submitted: April 17, 2007
First Posted: April 19, 2007
Results First Submitted: March 31, 2010
Results First Posted: May 3, 2010
Last Update Posted: April 15, 2014