A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration

Hereditary Angioedema Clinical Trial

— 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")

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00851409
• Other study ID #: C1 1207
• Status: Completed
• Phase: Phase 2
• Start date: June 2009
• Est. completion date: April 2010

Study information

• Verified date: April 2018
• Source: Pharming Technologies B.V.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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").

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: April 2010
• Est. primary completion date: March 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Genetic Diseases, Inborn
• Genetic Disorders
• Hereditary Angioedema

Intervention

Drug:
• Recombinant Human C1 Inhibitor
50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Locations

Country	Name	City	State
Netherlands	For information on sites, please contact Pharming Technologies	Leiden
Romania	Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department	Târgu-Mures

Sponsors (1)

Lead Sponsor	Collaborator
Pharming Technologies B.V.

Countries where clinical trial is conducted

Netherlands,  Romania, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	8 weeks
Secondary	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)	8 weeks