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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.
ClinicalTrials.gov Identifier:
NCT00262301
First received: December 1, 2005
Last updated: September 27, 2012
Last verified: September 2012
History of Changes
  Purpose

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 Intervention Phase
Hereditary Angioedema
Angioneurotic Edema
Genetic Disorders
 Drug: recombinant human C1 inhibitor
Drug: Placebo
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:

MedlinePlus related topics: Edema
U.S. FDA Resources

Further study details as provided by Pharming Technologies B.V.:

Primary Outcome Measures:
  • Time to Beginning of Relief of Symptoms [ Time Frame: up to 48 hours after study drug administration ] [ Designated as safety issue: No ]
    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:
  • Time to Minimal Symptoms [ Time Frame: up to 48 hours after study drug administration ] [ Designated as safety issue: No ]
    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.


Enrollment: 75
Study Start Date: June 2004
Study Completion Date: October 2009
Primary Completion Date: July 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: 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.
  More Information

Publications:

Additional 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
Study First Received: December 1, 2005
Results First Received: July 27, 2012
Last Updated: September 27, 2012
Health Authority: Netherlands: Independent Ethics Committee

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

ClinicalTrials.gov processed this record on May 21, 2013