A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration

NCT ID: NCT00851409

Last Updated: 2018-06-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-06-30
• Study Completion Date: 2010-04-30

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").

Conditions

Genetic Disorders; Hereditary Angioedema

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Recombinant Human C1 Inhibitor

Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor

Group Type: OTHER

Recombinant Human C1 Inhibitor

Intervention Type: DRUG

50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Interventions

Recombinant Human C1 Inhibitor

50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Intervention Type: DRUG

Other Intervention Names

"rhC1INH"; Ruconest

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pharming Technologies B.V.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bruno Giannetti, MD

Role: STUDY_CHAIR

COO Pharming

Locations

For information on sites, please contact Pharming Technologies

Leiden, , Netherlands

Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department

Târgu Mureş, , Romania

Countries

Netherlands; Romania

Other Identifiers

C1 1207

Identifier Type: -

Identifier Source: org_study_id