Clinical trial NCT01359969: Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients
Official title: Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of rhC1INH for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2-13 Years of Age
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema
Keywords: Hereditary Angioedema
HAE
Angioedema
Recombinant C1 Inhibitor
rhC1INH
Pediatrics
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2011-05-25
Enrollment: 57
Sex: ALL
Minimum age (years): 2
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed description: This study was an open-label, Phase 2, non-comparative, multinational, multicenter clinical study in pediatric patients from 2 up to and including 13 years of age, with a confirmed diagnosis of HAE. Patients were eligible for treatment with recombinant human C1-inhibitor (rhC1INH) if they presented to the clinic within 5 hours of onset with an acute attack of at least moderate severity without signs of spontaneous regression. Patients received rhC1INH at a dose of 50 U/kg body weight up to a maximum of 4200 U. The reconstituted solution was administered as a slow intravenous (iv) injection over approximately 5 minutes. The patients remained in hospital and were closely monitored in the study center for at least 4 hours after study medication administration.

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

Primary outcomes:
measure: 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.
timeFrame: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.
Secondary outcomes:
measure: 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
timeFrame: The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.
Other outcomes:
Locations:
city: Portland
country: United States
facility: Portland Clinical Research/AAIM Care, LLC
state: Oregon
city: Plzen
country: Czechia
facility: UIA FN Plzen ( Institute of Immunology and Allergology), Faculty Hospital Plzen
state: Alej Svobody 80
city: Prague
country: Czechia
facility: University Hospital Motol, Institute of Immunology
state: V Úvalu 84
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
state: Charitéplatz 1
city: Munich
country: Germany
facility: Klinikum Rechts der Isar, Technical University Munich
city: Budapest
country: Hungary
facility: Heim Pál Gyermekkórház, II. számú Gyermek Belgyógyászati Osztály
state: Madarász Utca 22-24
city: Haifa
country: Israel
facility: Bnei Zion Hospital
city: Tel Hashomer
country: Israel
facility: Sheba Medical Center
city: Tel-Aviv
country: Israel
facility: Souraski Medical Center
city: Milan
country: Italy
facility: Hospital Luigi Sacco
city: Salerno
country: Italy
facility: Azienda Ospedaliera Universitaria S. Giovanni di Dio e Ruggi d'Aragona
city: Skopje
country: North Macedonia
facility: University Clinic Of Dermatology Skopje
city: Krakow
country: Poland
facility: Pediatric Hospital
city: Lublin
country: Poland
facility: Pediatric Hospital
city: Targu Mures
country: Romania
facility: Mures County Clinical Hospital
city: Martin
country: Slovakia
facility: Klinika detí a dorastu, Univerzitna nemocnica Martin
state: Kollárova 2
Study officials:
affiliation: VP Clinical Research & Medical Affairs at Pharming
name: Anurag Relan, MD
role: STUDY_DIRECTOR
References:
citation: Reshef A, Grivcheva-Panovska V, Kessel A, Kivity S, Klimaszewska-Rembiasz M, Moldovan D, Farkas H, Gutova V, Fritz S, Relan A, Giannetti B, Magerl M. Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children. Pediatr Allergy Immunol. 2019 Aug;30(5):562-568. doi: 10.1111/pai.13065. Epub 2019 May 29.