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

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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.

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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Angioneurotic Edema Genetic Disorders Drug: recombinant human C1 inhibitor Drug: Placebo Phase 3

Detailed Description:
A prospectively planned interim analysis will be performed on the double-blind data.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 75 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 III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Study Start Date : June 2004
Actual Primary Completion Date : July 2009
Actual Study Completion Date : October 2009

Resource links provided by the National Library of Medicine

U.S. FDA Resources

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
Placebo Comparator: Saline
Saline solution
Drug: Placebo
IV
Other Names:
  • saline
  • physiological salt solution



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 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.


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 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.



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Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT00262301


Locations
Netherlands
For information on sites, please contact Pharming Medical Affairs Deparment
Leiden, Netherlands, 2300 AL
Romania
Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Tirgu Mures, Romania, 541103
Sponsors and Collaborators
Pharming Technologies B.V.
Investigators
Study Chair: Jan Nuijens, MD, PhD Pharming Group N.V.

Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT00262301     History of Changes
Other Study ID Numbers: C1 1304-01
First Posted: December 6, 2005    Key Record Dates
Results First Posted: August 30, 2012
Last Update Posted: October 2, 2012
Last Verified: September 2012

Additional relevant MeSH terms:
Angioedemas, Hereditary
Angioedema
Genetic Diseases, Inborn
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Complement C1 Inactivator Proteins
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs