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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Pharming Technologies B.V.
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.
ClinicalTrials.gov Identifier:
NCT01359969
First received: May 19, 2011
Last updated: March 12, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Hereditary Angioedema
Drug: rhC1INH
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Pharming Technologies B.V.:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
  • Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ] [ Designated as safety issue: Yes ]
  • Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ] [ Designated as safety issue: Yes ]
  • Immunogenicity by assessing IgE antibodies against rabbit epithelium [ Time Frame: 28 Days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Beginning of relief [ Time Frame: 24 Hours ] [ Designated as safety issue: No ]
    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

  • Minimal symptoms [ Time Frame: 24 Hours ] [ Designated as safety issue: No ]
    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

  • Complete resolution [ Time Frame: 28 Days (diary recording) ] [ Designated as safety issue: No ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved

  • Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ] [ Designated as safety issue: No ]
    C1INH activity

  • Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ] [ Designated as safety issue: No ]
    C4 levels


Estimated Enrollment: 20
Study Start Date: December 2011
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   2 Years to 13 Years
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01359969

Contacts
Contact: Annemarie Kleefstra +31 71 5247 400 a.kleefstra@pharming.com
Contact: Anurag Relan, MD a.relan@pharming.com

Locations
Germany
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         
Israel
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         
Italy
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         
Poland
Pediatric Hospital Recruiting
Krakow, Poland
Principal Investigator: Dr. Maria Klimaszewska-Rembiasz         
Pediatric Hospital Recruiting
Lublin, Poland
Principal Investigator: Prof. Andrzej Emeryk         
Romania
Mures County Clinical Hospital Recruiting
Targu Mures, Romania
Principal Investigator: Prof. dr. Dumitru Moldovan         
Sponsors and Collaborators
Pharming Technologies B.V.
  More Information

No publications provided

Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT01359969     History of Changes
Other Study ID Numbers: C1 1209, 2011-000987-92
Study First Received: May 19, 2011
Last Updated: March 12, 2014
Health Authority: Italy: The Italian Medicines Agency
Germany: Federal Institute for Drugs and Medical Devices
Poland: Ministry of Health
Romania: National Medicines Agency
Israel: Ministry of Health

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

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

ClinicalTrials.gov processed this record on July 22, 2014