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:
NCT00225147
First received: September 20, 2005
Last updated: February 20, 2013
Last verified: February 2013
  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 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 Intervention Phase
Hereditary Angioedema
Angioneurotic Edema
Drug: Recombinant Human C1 Inhibitor
Drug: placebo
Phase 2
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 II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Resource links provided by NLM:


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 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:
  • 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 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.


Enrollment: 77
Study Start Date: July 2005
Study Completion Date: January 2010
Primary Completion Date: October 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
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

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00225147

Locations
Netherlands
For information on sites please contact Pharming Medical Affairs Department
Leiden, Netherlands, 2300 AL
Sponsors and Collaborators
Pharming Technologies B.V.
Investigators
Study Director: Anurag Relan, MD 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: NCT00225147     History of Changes
Other Study ID Numbers: C1 1205-01
Study First Received: September 20, 2005
Results First Received: February 22, 2012
Last Updated: February 20, 2013
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

Additional relevant MeSH terms:
Angioedemas, Hereditary
Angioedema
Edema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Signs and Symptoms
Genetic Diseases, Inborn
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 July 28, 2014