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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.
ClinicalTrials.gov Identifier:
NCT00851409
First received: February 25, 2009
Last updated: December 20, 2012
Last verified: December 2012
  Purpose

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 Intervention Phase
Genetic Disorders
Hereditary Angioedema
Drug: Recombinant Human C1 Inhibitor
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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")

Resource links provided by NLM:


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

Primary Outcome Measures:
  • HAE Attacks/Week [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
    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:
  • The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters. [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
    "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)


Enrollment: 25
Study Start Date: June 2009
Study Completion Date: April 2010
Primary Completion Date: March 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: 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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
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: NCT00851409

Locations
Netherlands
For information on sites, please contact Pharming Technologies
Leiden, Netherlands
Romania
Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Tirgu Mures, Romania, 541103
Sponsors and Collaborators
Pharming Technologies B.V.
Investigators
Study Chair: Bruno Giannetti, MD COO Pharming
  More Information

No publications provided

Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT00851409     History of Changes
Other Study ID Numbers: C1 1207
Study First Received: February 25, 2009
Results First Received: November 9, 2012
Last Updated: December 20, 2012
Health Authority: Netherlands: Independent Ethics Committee

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

ClinicalTrials.gov processed this record on September 18, 2014