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

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ClinicalTrials.gov Identifier: NCT01359969
Recruitment Status : Completed
First Posted : May 25, 2011
Last Update Posted : July 18, 2018
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Drug: rhC1INH Phase 2

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 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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2 up to and Including 13 Years of Age
Actual Study Start Date : January 17, 2012
Actual Primary Completion Date : July 17, 2017
Actual Study Completion Date : July 17, 2017

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 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.
Drug: 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).
Other Name: Ruconest




Primary Outcome Measures :
  1. Number of patients with Adverse Events [ Time Frame: 90 Days ]
    The safety and tolerability is assessed by the number of patients with at least one adverse event.

  2. Number of patients with positive neutralizing C1INH and rhC1INH specific antibody tests [ Time Frame: 90 Days ]
    Immunogenicity by assessing antibodies against rhC1INH (IgG and IgM) anti-rhC1INH

  3. Number of patients with confirmed host related impurities antibodies (Anti-HRI) after exposure to rhC1INH [ Time Frame: 90 Days ]
    Immunogenicity by assessing antibodies against host related impurities (anti-HRI)

  4. Number of patients with positive IgE against rabbit epithelium after exposure to rhC1INH with negative IgE test prior to exposure [ Time Frame: 28 Days ]
    Immunogenicity by assessing IgE antibodies against rabbit epithelium


Secondary Outcome Measures :
  1. Time to beginning of relief [ Time Frame: 24 Hours ]
    Time to beginning of relief assessed by using the overall severity visual analog scale(VAS), defined as the first time point with a decrease of at least 20 mm with respect to baseline at any eligible location, with persistence at the next time point

  2. Time to minimal symptoms [ Time Frame: 24 Hours ]
    Time to minimal symptoms assessed by using the overall severity VAS, defined as the first time point at which the overall severity VAS falls below 20 mm for each assessed location

  3. Time to complete resolution [ Time Frame: 28 Days (diary recording) ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved

  4. Functional C1 Esterase Inhibitor activity 2-4 hours post dose [ Time Frame: 4 Hours ]
    For pharmacokinetic profile the plasma functional C1INH activity was measured at presentation, 5 minutes post dose, and 2 to 4 hours post-dose for the first acute attack treated in the study. Functional C1INH concentrations are in units % of normal, i.e. results were expressed as a percentage based on a pool of plasma collected from healthy subjects, which was originally set on 100%

  5. C4 concentration 2-4 hours post dose [ Time Frame: 4 Hours ]
    For pharmacodynamic evaluation plasma C4 was measured at presentation, 5 minutes post-dose and 2 to 4 hours post-dose for the first attack treated in the study.



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Ages Eligible for Study:   2 Years to 13 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 (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) IgE test

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01359969


Locations
United States, Oregon
Portland Clinical Research/AAIM Care, LLC
Portland, Oregon, United States, 97015
Czechia
UIA FN Plzen ( Institute of Immunology and Allergology), Faculty Hospital Plzen
Plzen, Alej Svobody 80, Czechia, 304 60 Plzen
University Hospital Motol, Institute of Immunology
Prague, V Úvalu 84, Czechia, 150 06 Prague
Germany
Charité - Universitätsmedizin Berlin
Berlin, Charitéplatz 1, Germany, 10117
Klinikum Rechts der Isar, Technical University Munich
Munich, Germany
Hungary
Heim Pál Gyermekkórház, II. számú Gyermek Belgyógyászati Osztály
Budapest, Madarász Utca 22-24, Hungary, 1131
Israel
Bnei Zion Hospital
Haifa, Israel
Sheba Medical Center
Tel Hashomer, Israel
Souraski Medical Center
Tel-Aviv, Israel
Italy
Hospital Luigi Sacco
Milan, Italy
Azienda Ospedaliera Universitaria S. Giovanni di Dio e Ruggi d'Aragona
Salerno, Italy, 84131
Macedonia, The Former Yugoslav Republic of
University Clinic Of Dermatology Skopje
Skopje, Macedonia, The Former Yugoslav Republic of, 1000
Poland
Pediatric Hospital
Krakow, Poland
Pediatric Hospital
Lublin, Poland
Romania
Mures County Clinical Hospital
Targu Mures, Romania
Slovakia
Klinika detí a dorastu, Univerzitna nemocnica Martin
Martin, Kollárova 2, Slovakia, 036 59 Martin
Sponsors and Collaborators
Pharming Technologies B.V.

Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT01359969     History of Changes
Other Study ID Numbers: C1 1209
2011-000987-92 ( EudraCT Number )
First Posted: May 25, 2011    Key Record Dates
Last Update Posted: July 18, 2018
Last Verified: July 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Pharming Technologies B.V.:
Hereditary Angioedema
HAE
Angioedema
Recombinant C1 Inhibitor
rhC1INH
Pediatrics

Additional relevant MeSH terms:
Complement C1 Inhibitor Protein
Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs