Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients

NCT ID: NCT01359969

Last Updated: 2024-03-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 57 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-17
• Study Completion Date: 2017-07-17

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.

Conditions

Hereditary Angioedema

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Recombinant Human C1 Inhibitor

Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.

Group Type: EXPERIMENTAL

rhC1INH

Intervention Type: DRUG

Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).

Interventions

rhC1INH

Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).

Intervention Type: DRUG

Other Intervention Names

Ruconest

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

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 13 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pharming Technologies B.V.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anurag Relan, MD

Role: STUDY_DIRECTOR

VP Clinical Research & Medical Affairs at Pharming

Locations

Portland Clinical Research/AAIM Care, LLC

Portland, Oregon, United States

UIA FN Plzen ( Institute of Immunology and Allergology), Faculty Hospital Plzen

Pilsen, Alej Svobody 80, Czechia

University Hospital Motol, Institute of Immunology

Prague, V Úvalu 84, Czechia

Charité - Universitätsmedizin Berlin

Berlin, Charitéplatz 1, Germany

Klinikum Rechts der Isar, Technical University Munich

Munich, , Germany

Heim Pál Gyermekkórház, II. számú Gyermek Belgyógyászati Osztály

Budapest, Madarász Utca 22-24, Hungary

Bnei Zion Hospital

Haifa, , Israel

Souraski Medical Center

Tel Aviv, , Israel

Sheba Medical Center

Tel Litwinsky, , Israel

Hospital Luigi Sacco

Milan, , Italy

Azienda Ospedaliera Universitaria S. Giovanni di Dio e Ruggi d'Aragona

Salerno, , Italy

University Clinic Of Dermatology Skopje

Skopje, , North Macedonia

Pediatric Hospital

Krakow, , Poland

Pediatric Hospital

Lublin, , Poland

Mures County Clinical Hospital

Târgu Mureş, , Romania

Klinika detí a dorastu, Univerzitna nemocnica Martin

Martin, Kollárova 2, Slovakia

Countries

United States; Czechia; Germany; Hungary; Israel; Italy; North Macedonia; Poland; Romania; Slovakia

References

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.

Reference Type: RESULT

PMID: 30993784 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2011-000987-92

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

C1 1209

Identifier Type: -

Identifier Source: org_study_id