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

This study is currently recruiting participants.
Verified February 2013 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: February 20, 2013
Last verified: February 2013
History of Changes

May 19, 2011
February 20, 2013
December 2011
January 2014   (final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT01359969 on ClinicalTrials.gov Archive Site
  • 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
  • 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 ]
  • 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
Not Provided
Not Provided
 
Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients
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

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.
Not Provided
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hereditary Angioedema
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
Experimental: Recombinant Human C1 Inhibitor
Intervention: Drug: rhC1INH
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
20
January 2014
January 2014   (final data collection date for primary outcome measure)

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
Both
2 Years to 13 Years
No
Contact: Annemarie Kleefstra +31 71 5247 400 a.kleefstra@pharming.com
Contact: Anurag Relan, MD a.relan@pharming.com
Germany,   Israel,   Italy,   Poland,   Romania
 
NCT01359969
C1 1209, 2011-000987-92
No
Pharming Technologies B.V.
Pharming Technologies B.V.
Not Provided
Not Provided
Pharming Technologies B.V.
February 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP