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A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)

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: NCT00851409
Recruitment Status : Completed
First Posted : February 26, 2009
Results First Posted : January 31, 2013
Last Update Posted : June 26, 2018
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

Study Description
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Brief Summary:
Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").

Condition or disease Intervention/treatment Phase
Genetic Disorders Hereditary Angioedema Drug: Recombinant Human C1 Inhibitor Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Exploratory Phase II Study of the Safety and Immunogenicity of Repeated "rhC1INH" Administration of 50 U/Kg in Patients With Hereditary C1 Inhibitor Deficiency ("HAE")
Study Start Date : June 2009
Actual Primary Completion Date : March 2010
Actual Study Completion Date : April 2010

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Recombinant Human C1 Inhibitor
Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
 Drug: Recombinant Human C1 Inhibitor
50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
Other Names:
  • "rhC1INH"
  • Ruconest


Outcome Measures
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Primary Outcome Measures :
  1. HAE Attacks/Week [ Time Frame: 8 weeks ]
    Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).


Secondary Outcome Measures :
  1. The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters. [ Time Frame: 8 weeks ]
    "PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Aged at least 18 years
  • Signed informed consent
  • Comfirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50% of normal, and/or proven HAE ,mutation in C1INH gene.

Exclusion Criteria:

  • A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs.
  • A history of allergic reactions to C1INH products or rabbit protein.
  • Any reported SAE related to study drug administration (withdrawal criterium)
  • Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
  • A diagnosis of acquired C1INH deficiency.
  • Woman of child bearing potential, pregnancy or breast-feeding
  • previous treatment within the last 3 months with plasma-derived C1INH
  • Any clinically significant abnormality in the routine haematology, biochemistry and urinalysis
  • Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives.
  • Any changes since screening that would exclude subject based on above exclusion criteria.
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): NCT00851409


Locations
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Netherlands
For information on sites, please contact Pharming Technologies
Leiden, Netherlands
Romania
Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Târgu-Mureş, Romania, 541103
Sponsors and Collaborators
Pharming Technologies B.V.
Investigators
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Study Chair: Bruno Giannetti, MD COO Pharming
More Information
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Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT00851409    
Other Study ID Numbers: C1 1207
First Posted: February 26, 2009    Key Record Dates
Results First Posted: January 31, 2013
Last Update Posted: June 26, 2018
Last Verified: April 2018
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Genetic Diseases, Inborn
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
 Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Immunologic Deficiency Syndromes
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs