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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: NCT00262301
Recruitment Status : Completed
First Posted : December 6, 2005
Results First Posted : August 30, 2012
Last Update Posted : October 2, 2012
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

Tracking Information
First Submitted Date  ICMJE December 1, 2005
First Posted Date  ICMJE December 6, 2005
Results First Submitted Date  ICMJE July 27, 2012
Results First Posted Date  ICMJE August 30, 2012
Last Update Posted Date October 2, 2012
Study Start Date  ICMJE June 2004
Actual Primary Completion Date July 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 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 has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
Original Primary Outcome Measures  ICMJE
 (submitted: December 5, 2005)
Time to the beginning of 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 time-points were: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to minimal symptoms has been calculated by using the exact time-points on which each assessment was performed.
Original Secondary Outcome Measures  ICMJE
 (submitted: December 5, 2005)
Time to complete resolution of angioedema symptoms
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 III Study of the Efficacy and Safety 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 leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Detailed Description A prospectively planned interim analysis will be performed on the double-blind data.
Study Type  ICMJE Interventional
Study Phase  ICMJE 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
  • Genetic Disorders
Intervention  ICMJE
  • Drug: recombinant human C1 inhibitor
    IV
    Other Names:
    • "rhC1INH"
    • Ruconest
    • conestat alfa
  • Drug: Placebo
    IV
    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
  • Placebo Comparator: Saline
    Saline solution
    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)
75
Original Enrollment  ICMJE
 (submitted: December 5, 2005)
50
Actual Study Completion Date  ICMJE October 2009
Actual Primary Completion Date July 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Clear clinical and laboratory diagnosis of HAE
  • Baseline plasma level of functional C1INH of less than 50% of normal
  • Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack

Exclusion Criteria:

  • Acquired angioedema
  • Pregnancy or breastfeeding
  • Participation in another clinical study within prior 3 months
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 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,   Romania
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00262301
Other Study ID Numbers  ICMJE C1 1304-01
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 Chair: Jan Nuijens, MD, PhD Pharming Group N.V.
PRS Account Pharming Technologies B.V.
Verification Date September 2012

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