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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.

Study Description
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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


Condition or disease Intervention/treatment Phase
Hereditary Angioedema Angioneurotic Edema Drug: Recombinant Human C1 Inhibitor Drug: placebo Phase 2 Phase 3

Detailed Description:
A prospectively planned interim analysis will be performed on the double-blind data.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 77 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : July 2005
Actual Primary Completion Date : October 2009
Actual Study Completion Date : January 2010

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 100 IU/kg rhC1INH
100 IU/kg Recombinant human C1 inhibitor
 Drug: Recombinant Human C1 Inhibitor
IV
Other Names:
  • "rhC1INH"
  • Ruconest
  • conestat alfa
Experimental: 50 IU/kg rhC1INH
50 IU/kg Recombinant human C1 inhibitor
 Drug: Recombinant Human C1 Inhibitor
IV
Other Names:
  • "rhC1INH"
  • Ruconest
  • conestat alfa
Placebo Comparator: Saline Drug: placebo
saline solution
Other Names:
  • saline
  • physiological salt solution


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00225147


Locations
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Netherlands
For information on sites please contact Pharming Medical Affairs Department
Leiden, Netherlands, 2300 AL
Sponsors and Collaborators
Pharming Technologies B.V.
Investigators
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Study Director: Anurag Relan, MD Pharming Group N.V.
More Information
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Publications of Results:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT00225147    
Other Study ID Numbers: C1 1205-01
First Posted: September 23, 2005    Key Record Dates
Results First Posted: August 31, 2012
Last Update Posted: February 22, 2013
Last Verified: February 2013
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
 Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs