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

This study is currently recruiting participants.
Verified October 2016 by Pharming Technologies B.V.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01359969
First Posted: May 25, 2011
Last Update Posted: October 17, 2016
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.
Information provided by (Responsible Party):
Pharming Technologies B.V.
May 19, 2011
May 25, 2011
October 17, 2016
December 2011
December 2017   (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 ]
  • Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing IgE antibodies against rabbit epithelium [ Time Frame: 28 Days ]
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 ]
Complete list of historical versions of study NCT01359969 on ClinicalTrials.gov Archive Site
  • 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
  • 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
  • Complete resolution [ Time Frame: 28 Days (diary recording) ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved
  • Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ]
    C1INH activity
  • Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ]
    C4 levels
  • Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ]
  • Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ]
  • 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
  • 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
  • Complete resolution [ Time Frame: 28 Days (diary recording) ]
    Patient-recorded time at which all angioedema symptoms at all locations have resolved
  • Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ]
    C1INH activity
  • Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ]
    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
Intervention Model: Single Group Assignment
Masking: None (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
December 2017
December 2017   (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
Sexes Eligible for Study: All
2 Years to 13 Years   (Child)
No
Contact: Tessa Heikamp +31 71 5247 425 t.heikamp@pharming.com
Contact: Anurag Relan, MD +31 71 5247 400 a.relan@pharming.com
Czech Republic,   Germany,   Hungary,   Israel,   Italy,   Macedonia, The Former Yugoslav Republic of,   Poland,   Romania,   Slovakia
 
 
NCT01359969
C1 1209
2011-000987-92 ( EudraCT Number )
No
Not Provided
Not Provided
Pharming Technologies B.V.
Pharming Technologies B.V.
Not Provided
Not Provided
Pharming Technologies B.V.
October 2016

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