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Trial record 75 of 9085 for:    Genetic Diseases, Inborn AND genetic disorder

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.

Tracking Information
First Submitted Date  ICMJE February 25, 2009
First Posted Date  ICMJE February 26, 2009
Results First Submitted Date  ICMJE November 9, 2012
Results First Posted Date  ICMJE January 31, 2013
Last Update Posted Date June 26, 2018
Study Start Date  ICMJE June 2009
Actual Primary Completion Date March 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 20, 2012)
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).
Original Primary Outcome Measures  ICMJE
 (submitted: February 25, 2009)
to evaluate the occurrence of HAE attacks under prophylactic administration of rC1INH (50 U/Kg, once a week)
Change History Complete list of historical versions of study NCT00851409 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 20, 2012)
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)
Original Secondary Outcome Measures  ICMJE
 (submitted: February 25, 2009)
to evaluate the pK parameters, safety and immunogenicity on repeated administration of rC1INH
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
Official Title  ICMJE 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")
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").
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Genetic Disorders
  • Hereditary Angioedema
Intervention  ICMJE 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
Study Arms  ICMJE Recombinant Human C1 Inhibitor
Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
Intervention: Drug: Recombinant Human C1 Inhibitor
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 25, 2009)
25
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE April 2010
Actual Primary Completion Date March 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Netherlands,   Romania
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00851409
Other Study ID Numbers  ICMJE C1 1207
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pharming Technologies B.V.
Study Sponsor  ICMJE Pharming Technologies B.V.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Bruno Giannetti, MD COO Pharming
PRS Account Pharming Technologies B.V.
Verification Date April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP