Phase 3
N=38
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary Angioedema · Angioneurotic Edema
Bottom Line
View on ClinicalTrials.gov: NCT00225147 ↗Enrolled (actual)
38
Serious AEs
4.0%
Results posted
Aug 2012
Primary outcome: Primary: Time to Beginning of Relief of Symptoms — 68; 122; 258; 62.5 minutes — p=0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Recombinant Human C1 Inhibitor (Drug); placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Pharming Technologies B.V.
- Primary completion
- Oct 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Beginning of Relief of Symptoms |
68; 122; 258; 62.5 | 0.001 sig |
| SECONDARY Time to Minimal Symptoms |
245; 246.5; 1101; 145 | 0.040 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 lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Funding Source - FDA OOPD
Eligibility Criteria
Main Inclusion Criteria:
- Clear clinical and laboratory diagnosis of HAE
- Plasma level of functional C1INH of less than 50% of normal
- Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack
Main Exclusion Criteria:
- Acquired angioedema
- Pregnancy or breastfeeding
- Treatment with any investigational drug within prior 30 days
- Body weight >120 kg
Data sourced from ClinicalTrials.gov (NCT00225147). 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.