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Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00225147
Recruitment Status : Completed
First Posted : September 23, 2005
Results First Posted : August 31, 2012
Last Update Posted : February 22, 2013
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

Tracking Information
First Submitted Date  ICMJE September 20, 2005
First Posted Date  ICMJE September 23, 2005
Results First Submitted Date  ICMJE February 22, 2012
Results First Posted Date  ICMJE August 31, 2012
Last Update Posted Date February 22, 2013
Study Start Date  ICMJE July 2005
Actual Primary Completion Date October 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 27, 2012)
Time to Beginning of Relief of Symptoms [ Time Frame: up to 48 hours after study drug administration ]
The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.
Original Primary Outcome Measures  ICMJE
 (submitted: September 21, 2005)
Relief of angioedema symptoms
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 27, 2012)
Time to Minimal Symptoms [ Time Frame: up to 48 hours after study drug administration ]
The time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score. Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm. Assessment timepoints were: baseline, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to minimal symtoms has been calculated by using the exact timepoints on which each assessment was performed.
Original Secondary Outcome Measures  ICMJE
 (submitted: September 21, 2005)
Safety and tolerability; pharmacokinetics/pharmacodynamics
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Official Title  ICMJE A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Brief Summary

Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.

Funding Source - FDA OOPD

Detailed Description A prospectively planned interim analysis will be performed on the double-blind data.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Hereditary Angioedema
  • Angioneurotic Edema
Intervention  ICMJE
  • Drug: Recombinant Human C1 Inhibitor
    IV
    Other Names:
    • "rhC1INH"
    • Ruconest
    • conestat alfa
  • Drug: placebo
    saline solution
    Other Names:
    • saline
    • physiological salt solution
Study Arms  ICMJE
  • Experimental: 100 IU/kg rhC1INH
    100 IU/kg Recombinant human C1 inhibitor
    Intervention: Drug: Recombinant Human C1 Inhibitor
  • Experimental: 50 IU/kg rhC1INH
    50 IU/kg Recombinant human C1 inhibitor
    Intervention: Drug: Recombinant Human C1 Inhibitor
  • Placebo Comparator: Saline
    Intervention: Drug: placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 30, 2010)
77
Original Enrollment  ICMJE
 (submitted: September 21, 2005)
39
Actual Study Completion Date  ICMJE January 2010
Actual Primary Completion Date October 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  • Clear clinical and laboratory diagnosis of HAE
  • Plasma level of functional C1INH of less than 50% of normal
  • Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack

Main Exclusion Criteria:

  • Acquired angioedema
  • Pregnancy or breastfeeding
  • Treatment with any investigational drug within prior 30 days
  • Body weight >120 kg
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Netherlands
Removed Location Countries United States
 
Administrative Information
NCT Number  ICMJE NCT00225147
Other Study ID Numbers  ICMJE C1 1205-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pharming Technologies B.V.
Study Sponsor  ICMJE Pharming Technologies B.V.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Anurag Relan, MD Pharming Group N.V.
PRS Account Pharming Technologies B.V.
Verification Date February 2013

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