Hereditary Angioedema
Conditions
Keywords
C1 Inhibitor, Hereditary angioedema, Acute HAE attack
Brief summary
Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of involvement of the larynx.The planned extension study is designed to enrol subjects that participated in the pivotal study in order to provide them with C1-INH for treatment of acute HAE attacks for 24 months or until the licensing procedure for C1-INH is finalized, whatever comes first.
Interventions
Lyophilisate containing approximately 500 U C1-INH to be reconstituted with 10 mL water for injection; Single Dose: 20 U/kg b.w. iv
Sponsors
CSL Behring
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Documented congenital C1-INH deficiency * Acute HAE attack * Participation in base study CE1145\_3001 (NCT00168103) Key
Exclusion criteria
* Acquired angioedema * Treatment with any other investigational drug besides CE1145 in the last 30 days before study entry
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) | Up to 24 h after start of study treatment | The start of symptom relief was determined by subject self-assessment. |
| Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population) | Up to 24 h after start of study treatment | The start of symptom relief was determined by subject self-assessment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Complete Resolution of All HAE Symptoms (ITT Subject Population) | Up to Day 9 following an attack | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. |
| Time to Complete Resolution of All HAE Symptoms (ITT Attack Population) | Up to Day 9 following an attack | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. |
Countries
Canada, United States
Participant flow
Recruitment details
This was a multicenter, open-label extension study enrolling subjects at 15 sites in North America that had participated in study CE1145\_3001 (NCT00168103). Enrollment occurred between August 2005 and January 2008.
Participants by arm
| Arm | Count |
|---|---|
| C1 Esterase Inhibitor | 57 |
| Total | 57 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Lost to Follow-up | 13 |
| Overall Study | Other | 3 |
| Overall Study | Withdrawal by Subject | 22 |
Baseline characteristics
| Characteristic | C1 Esterase Inhibitor |
|---|---|
| Age, Continuous | 31.9 years STANDARD_DEVIATION 11.98 |
| Sex: Female, Male Female | 38 Participants |
| Sex: Female, Male Male | 19 Participants |
| Type of HAE Type I HAE | 49 participants |
| Type of HAE Type II HAE | 7 participants |
| Type of HAE Unknown | 1 participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 25 / 57 |
| serious Total, serious adverse events | 1 / 57 |
Outcome results
Primary
Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population)
The start of symptom relief was determined by subject self-assessment.
Time frame: Up to 24 h after start of study treatment
Population: Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| C1 Esterase Inhibitor | Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) | 0.46 hours |
Primary
Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population)
The start of symptom relief was determined by subject self-assessment.
Time frame: Up to 24 h after start of study treatment
Population: ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| C1 Esterase Inhibitor | Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population) | 0.37 hours |
Secondary
Time to Complete Resolution of All HAE Symptoms (ITT Attack Population)
Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card.
Time frame: Up to Day 9 following an attack
Population: ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| C1 Esterase Inhibitor | Time to Complete Resolution of All HAE Symptoms (ITT Attack Population) | 14.28 hours |
Secondary
Time to Complete Resolution of All HAE Symptoms (ITT Subject Population)
Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card.
Time frame: Up to Day 9 following an attack
Population: Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| C1 Esterase Inhibitor | Time to Complete Resolution of All HAE Symptoms (ITT Subject Population) | 15.48 hours |