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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01359969
Recruitment Status : Recruiting
First Posted : May 25, 2011
Last Update Posted : October 17, 2016
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

Study Type : Interventional  (Clinical Trial)
Estimated 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
Study Start Date : December 2011
Estimated Primary Completion Date : December 2017
Estimated Study Completion Date : December 2017

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Recombinant Human C1 Inhibitor 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. The primary objective is the assessment of safety and tolerability (adverse events, physical examination, vital signs, immunological and routine laboratory analyses et cetera) [ Time Frame: 90 Days ]
  2. Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ]
  3. Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ]
  4. Immunogenicity by assessing IgE antibodies against rabbit epithelium [ Time Frame: 28 Days ]

Secondary Outcome Measures :
  1. 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. 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. Complete resolution [ Time Frame: 28 Days (diary recording) ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved

  4. Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ]
    C1INH activity

  5. Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ]
    C4 levels

Information from the National Library of Medicine

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

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 identifier (NCT number): NCT01359969

Contact: Tessa Heikamp +31 71 5247 425
Contact: Anurag Relan, MD +31 71 5247 400

UIA FN Plzen ( Institute of Immunology and Allergology), Faculty Hospital Plzen Recruiting
Plzen, Alej Svobody 80, Czechia, 304 60 Plzen
Contact: Vaclava Gutova, Dr.    420 377 103 396   
University Hospital Motol, Institute of Immunology Recruiting
Prague, V Úvalu 84, Czechia, 150 06 Prague
Contact: Radana Zachova, Dr.    420 224 433 734   
Charité - Universitätsmedizin Berlin Recruiting
Berlin, Charitéplatz 1, Germany, 10117
Principal Investigator: Prof. Dr. Marcus Maurer         
Klinikum Rechts der Isar, Technical University Munich Recruiting
Munich, Germany
Principal Investigator: Prof. Dr. Murat Bas         
Heim Pál Gyermekkórház, II. számú Gyermek Belgyógyászati Osztály Not yet recruiting
Budapest, Madarász Utca 22-24, Hungary, 1131
Contact: Ferenc Fekete, Dr.    +36 70 365 8538   
Principal Investigator: Ferenc Fekete, Dr.         
Principal Investigator: Henriette Farkas, Prof.         
Bnei Zion Hospital Recruiting
Haifa, Israel
Principal Investigator: Dr. Aharon Kessel         
Sheba Medical Center Recruiting
Tel Hashomer, Israel
Principal Investigator: Dr. Avner Reshef         
Souraski Medical Center Recruiting
Tel-Aviv, Israel
Principal Investigator: Prof. Dr. Shmuel Kivity         
Hospital Luigi Sacco Recruiting
Milan, Italy
Principal Investigator: Prof. Dr. Marco Cicardi         
Azienda Ospedaliera Universitaria S. Giovanni di Dio e Ruggi d'Aragona Recruiting
Salerno, Italy, 84131
Principal Investigator: Prof. Dr. Massimo Triggiani         
Macedonia, The Former Yugoslav Republic of
University Clinic Of Dermatology Skopje Recruiting
Skopje, Macedonia, The Former Yugoslav Republic of, 1000
Contact: Prof. dr. Vesna Grivcheva Panovska, Prof. dr.    0038 92 31 22 265   
Pediatric Hospital Recruiting
Krakow, Poland
Principal Investigator: Dr. Maria Klimaszewska-Rembiasz         
Pediatric Hospital Recruiting
Lublin, Poland
Principal Investigator: Prof. Andrzej Emeryk         
Mures County Clinical Hospital Recruiting
Targu Mures, Romania
Principal Investigator: Prof. dr. Dumitru Moldovan         
Klinika detí a dorastu, Univerzitna nemocnica Martin Recruiting
Martin, Kollárova 2, Slovakia, 036 59 Martin
Contact: Milos Jesenak, Prof. Dr.    421 434 203 254   
Sponsors and Collaborators
Pharming Technologies B.V.

Responsible Party: Pharming Technologies B.V. 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: October 17, 2016
Last Verified: October 2016

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

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