Phase 2
Completed N=57
Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients
Source: ClinicalTrials.gov NCT01359969 ↗Enrolled (actual)
57
Serious AEs
15.0%
Results posted
Mar 2024
Primary outcomePrimary: Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief. — 60 minutes
Summary
This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief. |
60 | — |
| SECONDARY Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations |
122.5 | — |
Eligibility Criteria
Inclusion Criteria
- From 2 up to and including 13 years of age
- Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal)
- Signed written Informed Consent Form (ICF)(parental permission) signed by the legal guardian(s)
- Clinical symptoms of an acute HAE attack
- Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred
- Attack severity moderate or greater, as rated by the investigator
Exclusion Criteria
- A diagnosis of acquired C1INH deficiency (AAE)
- A medical history of allergy to rabbits or rabbit-derived products or positive anti-rabbit epithelium (dander) immunoglobuline E (IgE) test
Data sourced from ClinicalTrials.gov (NCT01359969). 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. Informational only — not medical advice.