- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00851409
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)
April 3, 2018 updated by: Pharming Technologies B.V.
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")
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").
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
25
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Leiden, Netherlands
- For information on sites, please contact Pharming Technologies
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Târgu-Mureş, Romania, 541103
- Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Other: Recombinant Human C1 Inhibitor
Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
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50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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HAE Attacks/Week
Time Frame: 8 weeks
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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).
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8 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
Time Frame: 8 weeks
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"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)
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8 weeks
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Chair: Bruno Giannetti, MD, COO Pharming
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
June 1, 2009
Primary Completion (Actual)
March 1, 2010
Study Completion (Actual)
April 1, 2010
Study Registration Dates
First Submitted
February 25, 2009
First Submitted That Met QC Criteria
February 25, 2009
First Posted (Estimate)
February 26, 2009
Study Record Updates
Last Update Posted (Actual)
June 26, 2018
Last Update Submitted That Met QC Criteria
April 3, 2018
Last Verified
April 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Skin Diseases
- Immunologic Deficiency Syndromes
- Immune System Diseases
- Hypersensitivity, Immediate
- Skin Diseases, Vascular
- Hypersensitivity
- Urticaria
- Hereditary Complement Deficiency Diseases
- Primary Immunodeficiency Diseases
- Genetic Diseases, Inborn
- Angioedema
- Angioedemas, Hereditary
- Physiological Effects of Drugs
- Immunosuppressive Agents
- Immunologic Factors
- Complement Inactivating Agents
- Complement C1 Inhibitor Protein
Other Study ID Numbers
- C1 1207
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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