A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration

Phase 2

Completed

• Conditions: Genetic Disorders; Hereditary Angioedema
• Interventions: Drug: Recombinant Human C1 Inhibitor

• Registration Number: NCT00851409
• Lead Sponsor: Pharming Technologies B.V.

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 available

Study Timeline

• Study First Submitted: 2009-02-25
• Study First Submitted QC: 2009-02-25
• Study First Posted: 2009-02-26
• Study Start: 2009-06
• Primary Completion: 2010-03
• Study Completion: 2010-04
• Results First Submitted: 2012-11-09
• Results First Submitted QC: 2012-12-20
• Results First Posted: 2013-01-31
• Status Verified: 2018-04
• Last Update Submitted: 2018-04-03
• Last Update Posted: 2018-06-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Recombinant Human C1 Inhibitor	Recombinant Human C1 Inhibitor	Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor

Primary Outcome Measures

Name	Time	Method
HAE Attacks/Week	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).

Secondary Outcome Measures

Name	Time	Method
The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.	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)

Trial Locations

Locations (2)

Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department; 🇷🇴 Târgu-Mureş, Romania

For information on sites, please contact Pharming Technologies; 🇳🇱 Leiden, Netherlands