Phase 3
N=32
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary Angioedema · Angioneurotic Edema · Genetic Disorders
Bottom Line
View on ClinicalTrials.gov: NCT00262301 ↗Enrolled (actual)
32
Serious AEs
2.3%
Results posted
Aug 2012
Primary outcome: Primary: Time to Beginning of Relief of Symptoms — 62; 508; 61 minutes — p=0.003
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- recombinant human C1 inhibitor (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Pharming Technologies B.V.
- Primary completion
- Jul 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Beginning of Relief of Symptoms |
62; 508; 61 | 0.003 sig |
| SECONDARY Time to Minimal Symptoms |
480; 1440; 241 | 0.005 sig |
Summary
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Eligibility Criteria
Inclusion Criteria
- Clear clinical and laboratory diagnosis of HAE
- Baseline plasma level of functional C1INH of less than 50% of normal
- Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack
Exclusion Criteria
- Acquired angioedema
- Pregnancy or breastfeeding
- Participation in another clinical study within prior 3 months
Data sourced from ClinicalTrials.gov (NCT00262301). 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.