A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)
This study has been completed.
Sponsor:
Pharming Technologies B.V.
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
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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:
Genetics Home Reference related topics:
hereditary angioedema
Drug Information available for:
Conestat alfa
U.S. FDA Resources
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:
|
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
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 Genetic Diseases, Inborn Vascular Diseases Cardiovascular Diseases Urticaria Skin Diseases, Vascular Skin Diseases Hypersensitivity, Immediate Hypersensitivity |
Immune System Diseases Complement C1 Complement C1 Inhibitor Protein Complement C1 Inactivator Proteins Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Complement Inactivating Agents Immunosuppressive Agents |
ClinicalTrials.gov processed this record on June 18, 2013