Phase 3
N=113
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT00438815 ↗Enrolled (actual)
113
Serious AEs
0.0%
Results posted
Jun 2010
Primary outcome: Primary: Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf — 609; 598 attacks
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- C1 esterase inhibitor [human] (C1INH-nf) (Biological)
- Age
- Pediatric, Adult, Older Adult · 1+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Mar 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf |
609; 598 | — |
| PRIMARY Percent of HAE Attacks With Substantial Relief of the Defining Symptom |
87; 95 | — |
| SECONDARY Time to Beginning of Substantial Relief of the Defining Symptom |
0.75 | — |
| SECONDARY Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments |
1.50; 0.50; 0.50; 0.50; 0.75; 0.50 | — |
| SECONDARY Antigenic C1 Inhibitor (C1INH) Serum Levels |
10.7; 9.6 | — |
| SECONDARY Functional C1INH Serum Levels |
27.0; 39.2 | — |
| SECONDARY Complement C4 Serum Levels |
5.3; -0.2 | — |
Summary
The study objective was to evaluate the safety and efficacy of repeat use of C1INH-nf for the treatment of acute HAE attacks.
Eligibility Criteria
Inclusion Criteria
This study was open to all subjects who:
- Completed participation in LEVP2005-1/A (NCT00289211) and were not participating in LEVP2005-1/B (NCT01005888), any time after the 3-day telephone follow-up
- Completed participation in LEVP2005-1/B any time after the final prophylactic therapy in Part B
- Were enrolled but not randomized in LEVP2005-1/A after Part A was closed
- Were excluded from LEVP2005-1 for any of the following reasons:
- Pregnancy or lactation
- Age less than 6 years
- Narcotic addiction
- Presence of anti-C1INH autoantibodies
- Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:
- Had a diagnosis of HAE: evidence of a low C4 level plus either a low C1INH antigenic level or a low C1INH functional level, or
- Had a known HAE-causing C1INH mutation, or
- Had a diagnosis of HAE based on a strong family history of HAE as determined by the principal investigator
Exclusion Criteria
- History of allergic reaction to C1INH or other blood products
- Participated in any other investigational drug study within the past 30 days
- Received blood or a blood product in the past 60 days other than C1INH-nf
Data sourced from ClinicalTrials.gov (NCT00438815). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.