Attacks associated with hereditary angioedema (HAE) in pediatric population 2-13 years of age. MedDRA version: 18.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Ruconest
Product Name: Ruconest
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: CONESTAT ALFA
CAS Number: 80295-38-1
Concentration unit: U unit(s)
Concentratio
Sponsors
Pharming Technologies B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Screening • From 2 up to and including 13 years of age • Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Screening • A diagnosis of acquired C1INH deficiency (AAE) • A medical history of allergy to rabbits or rabbit-derived products (including rhC1INH, antisera), or positive anti-rabbit epithelium (dander) IgE test (cut off>0.35 kU/L in ImmunoCap® assay (Phadia, Sweden) or equivalent) • Treatment with investigational drug in another clinical study in the last 30 days • Any clinical significant abnormality in the physical examination and/or the routine laboratory assessments, that in the opinion of the Investigator makes the patient unsuitable for participation in the study • Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives Treatment • Any changes since screening that would exclude patient based on above exclusion criteria. • 10 HAE attacks were previously treated with study medication. • Suspicion for an alternate explanation of the symptoms other than an acute HAE attack. • Use of any disallowed concomitant medication since onset of acute HAE attack
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the clinical safety, immunogenicity and tolerability of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.;Secondary Objective: To assess the pharmacokinetics (PK) and pharmacodynamics (PD) of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients. To assess the efficacy of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients. ;Primary end point(s): Assessment of safety and tolerability.;Timepoint(s) of evaluation of this end point: This will be evaluated by continuous recording adverse events. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Time to beginning of relief, time to minimal symptoms, time to complete resolution. Pharmacokinetic and pharmacodynamic parameters (C1INH activity and C4 in plasma) during treatment for the first attack.;Timepoint(s) of evaluation of this end point: The clinical endpoints will be evaluated following the treatment of an attack. Blood samples for PK and PD will be taken at 5 minutes and between 2-4 hours after the Ruconest injection at the first attack. | — |
Countries
Czech Republic, Germany, Hungary, Israel, Italy, Slovakia
Contacts
Public ContactTessa Heimap - van Rossenberg
Pharming Technologies B.V.
Outcome results
None listed