Back to resultsA Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)
Primary Purpose
Genetic Disorders, Hereditary Angioedema
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Recombinant Human C1 Inhibitor
Sponsored by
About this trial
This is an interventional treatment trial for Genetic Disorders
Eligibility 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.
Sites / Locations
- For information on sites, please contact Pharming Technologies
- Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Recombinant Human C1 Inhibitor
Arm Description
Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
Outcomes
Primary Outcome Measures
HAE Attacks/Week
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.
"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)
Full Information
NCT ID
NCT00851409
First Posted
February 25, 2009
Last Updated
April 3, 2018
Sponsor
Pharming Technologies B.V.
1. Study Identification
Unique Protocol Identification Number
NCT00851409
Brief Title
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
Acronym
OPERA
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 Type
Interventional
2. Study Status
Record Verification Date
April 2018
Overall Recruitment Status
Completed
Study Start Date
June 2009 (undefined)
Primary Completion Date
March 2010 (Actual)
Study Completion Date
April 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pharming Technologies B.V.
4. Oversight
Data Monitoring Committee
No
5. Study Description
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").
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Genetic Disorders, Hereditary Angioedema
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Recombinant Human C1 Inhibitor
Arm Type
Other
Arm Description
Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
Intervention Type
Drug
Intervention Name(s)
Recombinant Human C1 Inhibitor
Other Intervention Name(s)
"rhC1INH", Ruconest
Intervention Description
50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
Primary Outcome Measure Information:
Title
HAE Attacks/Week
Description
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).
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
Description
"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)
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bruno Giannetti, MD
Organizational Affiliation
COO Pharming
Official's Role
Study Chair
Facility Information:
Facility Name
For information on sites, please contact Pharming Technologies
City
Leiden
Country
Netherlands
Facility Name
Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
City
Târgu-Mureş
ZIP/Postal Code
541103
Country
Romania
12. IPD Sharing Statement
Learn more about this trial
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
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